Myopathy

This study is testing the meningococcal B vaccine (Bexsero) in children and young adults aged 2 to 25 who have autoimmune rheumatic diseases like juvenile arthritis or lupus. Researchers want to see if the vaccine triggers a strong immune response and if it is safe, especially fo…

This study tests whether the Butantan-Dengue vaccine is safe and works well in people aged 12–59 with autoimmune rheumatic diseases (like rheumatoid arthritis or lupus) who are stable on low or no immune-suppressing drugs. Participants receive one vaccine dose and are monitored f…

This study tests the safety and immune response of the 20-valent pneumococcal vaccine (PCV20) in children, adolescents, and young adults aged 2-25 with autoimmune rheumatic diseases like juvenile arthritis. All 114 participants will receive one dose of PCV20, and researchers will…

This study tests whether omega-3 fatty acid supplements from fish oil, combined with a healthy diet, can reduce disease activity in adults with dermatomyositis, a rare autoimmune disease causing muscle weakness and skin rashes. About 300 participants will take either fish oil cap…

This study follows about 300 people with Duchenne muscular dystrophy who are taking the drug givinostat as part of their normal care. Researchers will track safety issues like low platelet counts and bleeding, and see how patients' muscle function changes over up to 5 years. The …

This Phase 3 trial tests whether DYNE-101 can improve muscle function and daily life in 150 adults with myotonic dystrophy type 1 (DM1). Participants receive either the drug or a placebo by IV every few weeks for 48 weeks. The study measures how quickly people can stand from a ch…

This study tests a one-time gene therapy called RGX-202 in boys with Duchenne muscular dystrophy (DMD). The therapy delivers a mini version of the missing dystrophin protein to muscle cells. Researchers will check safety and whether it improves muscle function, like standing and …

This early-phase trial is testing a new drug called MTS109, made with mRNA technology, for people with moderate to severe autoimmune diseases like lupus and scleroderma. Fifteen participants will receive several injections over a month to see if it is safe and tolerable. The goal…

This program provides access to an experimental drug, AOC 1044, for people with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping exon 44. The goal is to help control the disease and improve muscle function. Participants must be at least 2 years …

This study tests a single intravenous dose of a gene therapy called GNR-097 in boys aged 4 to 9 with Duchenne muscular dystrophy (DMD). The therapy uses a harmless virus to deliver a shortened version of the dystrophin gene, which is missing or faulty in DMD. The trial aims to se…

This early-stage trial tests a new treatment called AZD0120 for adults with three autoimmune diseases: systemic sclerosis, idiopathic inflammatory myopathies, or difficult-to-treat rheumatoid arthritis. AZD0120 is made from a patient's own immune cells, which are modified in a la…

This Phase 1 trial tests the safety and tolerability of a new drug called AZD5492 in 72 adults with lupus, myositis, or rheumatoid arthritis. Participants receive one or two doses under the skin and are monitored for side effects and how the drug moves through the body. The study…

This early-stage study tests a new gene-editing medicine called PBGENE-DMD in 18 boys aged 2 to 7 with Duchenne muscular dystrophy. The treatment aims to correct the genetic mistake that causes the disease, potentially restoring muscle strength. Researchers are first checking if …

This study tests a one-time gene therapy called delandistrogene moxeparvovec in 83 people with Duchenne muscular dystrophy. The goal is to see if it is safe and helps the body make dystrophin, a protein missing in Duchenne. The trial is now enrolling non-ambulatory participants (…

This early-stage study tests a new drug (GSK5926371) in people with lupus and similar autoimmune diseases. The drug is designed to target and calm specific immune cells that attack the body. The main goal is to check if the drug is safe and how the body handles it, with 54 adults…

This early-stage trial tests a brain-computer interface called BrainGate for people with paralysis from conditions like ALS or spinal cord injury. A small sensor is placed in the brain to interpret movement-related signals, allowing users to control a tablet computer just by thin…

This phase 3 trial tests whether a new drug called ABP 938 works as well as the approved treatment Eylea HD for wet age-related macular degeneration, a leading cause of vision loss. About 304 people aged 50 and older with untreated wet AMD will receive injections of either drug i…

This study tests a single dose of SGT-003, a gene therapy that delivers a working version of the dystrophin gene to muscle cells. About 60 boys with Duchenne muscular dystrophy, aged from infancy to under 18, will receive the treatment and be followed for 5 years. The goal is to …

This early-stage study tests a new treatment called ALLO-329 for people with lupus, myositis, or scleroderma. The therapy uses specially engineered immune cells from a donor to target and calm the overactive immune system. The main goals are to check safety and see if it can impr…

This study is for people who have both an autoimmune disease (like rheumatoid arthritis, lupus, or multiple sclerosis) and an advanced cancer that has spread or cannot be removed by surgery. It tests the safety and effectiveness of the immunotherapy drug nivolumab, given alone or…

This study tests an oral drug called SAT-3247 in 51 boys aged 7 to 10 with Duchenne muscular dystrophy who can still walk. The goal is to find the best dose, check safety, and see if it helps muscle strength. Participants take the drug or a placebo for 12 weeks, and all continue …

This trial investigates whether using a device that sends mild electrical pulses to the thigh muscles (neuromuscular electrical stimulation, or NMES) can prevent muscle loss and weakness after abdominal surgery for colorectal or gastric cancer. Some participants will also receive…

This early-stage trial tests a single infusion of CRISPR-edited immune cells (CTX112) from a healthy donor in 80 adults with severe lupus, scleroderma, or myositis that hasn't responded to standard treatments. The goal is to see if the cells are safe and can reduce disease activi…

This study tests a drug called ublituximab for people with a rare autoimmune disease that causes severe muscle weakness. The goal is to see if adding this drug to standard treatment improves muscle strength and function more than standard treatment alone. About 30 adults with ear…

This early-phase study tests a new drug called OM336 in 39 adults with active Sjogren's disease or idiopathic inflammatory myopathy who have not improved with at least two prior treatments. OM336 is an engineered antibody designed to target and potentially reduce harmful immune c…

This early-stage study tests a single injection of a gene therapy called INS1201, given into the spinal fluid of young boys (ages 2 to 5) with Duchenne muscular dystrophy who can still walk. The main goal is to check if the treatment is safe and to see how it spreads in the body.…

This study tests a one-time treatment called CABA-201, made from a patient's own immune cells, for people with active inflammatory muscle diseases like dermatomyositis. The therapy aims to reset the immune system by targeting and removing certain immune cells. Researchers will ch…

This early-stage trial tests the safety and tolerability of a new drug called VX-670 in 52 adults with myotonic dystrophy type 1 (DM1), a genetic condition that causes muscle weakness and other problems. Participants receive either VX-670 or a placebo, and researchers will monito…

This study tests a small, refillable implant placed in the eye that slowly releases medication for wet age-related macular degeneration (nAMD). It aims to see if refilling the implant every 24 weeks works as well as getting a standard eye injection every 4 weeks. About 68 Chinese…

This Phase 3 study tests whether the drug mavorixafor can reduce serious infections and increase neutrophil levels in people with chronic neutropenia—a condition where the body doesn't make enough infection-fighting white blood cells. About 176 participants will receive either ma…

This study tests a new treatment called NKX019 for people with autoimmune diseases like systemic sclerosis, rheumatoid arthritis, and vasculitis. NKX019 uses specially engineered immune cells to target and calm overactive B cells that cause the disease. The goal is to see if it i…

This study tests whether a 4-month structured telerehabilitation program, delivered via video calls and an app, can reduce deaths and hospital readmissions in heart patients who have Post-Intensive Care Syndrome (PICS). About 326 adults aged 30-75 who were in the ICU for at least…

This early-stage trial is testing a new treatment called CC-97540, which uses a patient's own immune cells modified to target and destroy faulty immune cells. It is for people with severe forms of lupus, myositis, scleroderma, or rheumatoid arthritis that have not improved with s…

This study tests whether JAK inhibitors work better than calcineurin inhibitors as the first treatment for lung scarring in people with antisynthetase syndrome. About 80 adults with active lung disease will be followed for 12 months to see which drug improves breathing and surviv…

This Phase 3 trial tests whether baricitinib, a JAK inhibitor pill, can improve symptoms of dermatomyositis (a rare autoimmune disease causing muscle weakness and skin rashes) while allowing patients to stop steroids. 62 adults with active disease will receive baricitinib or a pl…

This Phase 3 trial tests a drug called dazukibart in people with active dermatomyositis or polymyositis, rare diseases that cause muscle weakness and inflammation. About 318 adults will receive either the drug or a placebo by IV every 4 weeks for 48 weeks. The study aims to see i…

This study tests a one-time gene therapy called SGT-003 in 80 boys with Duchenne muscular dystrophy who can still walk. The goal is to see if it helps them move better, like standing up faster and climbing stairs. Participants will receive either the therapy or a placebo first, t…

This early-stage study tests a new drug, GT801, in 22 adults with moderate to severe autoimmune diseases that haven't responded to standard treatments. The main goals are to check safety and find the right dose. Researchers will also look for signs that the drug helps control the…

This study tests the safety and feasibility of the BrainGate2 system, a brain-computer interface. Tiny sensors are placed in the brain area that controls movement, allowing people with tetraplegia (paralysis of all four limbs) to control a computer cursor or other assistive devic…

This study tests a gene therapy called SAR446268 for people aged 10 to 55 with myotonic dystrophy type 1. The therapy is given once through an IV and aims to reduce harmful DMPK RNA and improve muscle function. The trial has two parts: first, finding the safest dose in a small gr…

This study tests whether ruxolitinib, a drug that blocks a specific immune signal (interferon-γ), can help people with inclusion body myositis, a rare muscle disease that causes progressive weakness. About 80 adults aged 45 and older will receive either the drug or a placebo. The…

This phase 3 trial tests an experimental drug called AOC 1020 in 200 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes progressive muscle weakness. Participants receive either the drug or a placebo by IV infusion. The study measures muscle…

This study tests anifrolumab, a weekly injection, for adults with moderate to severe polymyositis or dermatomyositis—rare diseases that cause muscle weakness and inflammation. About 240 participants will receive either anifrolumab or a placebo added to their usual care for 52 wee…

This early-phase trial tests a new gene therapy called ASP2957 for X-linked myotubular myopathy (XLMTM), a rare muscle disease present at birth that often requires a ventilator to breathe. The therapy delivers a healthy copy of the MTM1 gene using a modified virus. Nine boys up t…

This early-stage study tests a new treatment made from healthy donor immune cells (CAR T cells) for people with severe autoimmune diseases like lupus that haven't responded to standard therapies. The goal is to see if these cells can safely target and calm the overactive immune s…

This early-phase trial tests a new drug called GT801 in 22 people with moderate-to-severe autoimmune diseases that haven't responded to other treatments. The main goal is to check safety and find the right dose. Researchers will also look for signs that the drug helps control the…

This study tests a new medicine called DYNE-251 in boys aged 4 to 18 with Duchenne muscular dystrophy (DMD) who can still walk. The goal is to see if it helps them move better and slows muscle damage. Participants will receive either the drug or a placebo every 4 weeks for about …

This early-phase trial is testing a new biologic drug called XmAb657 in 60 people, including healthy volunteers and those with autoimmune diseases like scleroderma, Sjögren's disease, and idiopathic inflammatory myopathies. The main goal is to check safety and how the body handle…

This early-stage study is testing a new cell therapy called FT819 for people with severe autoimmune diseases like lupus, vasculitis, and scleroderma. The treatment involves giving specially engineered immune cells along with chemotherapy drugs. The main goal is to see if it's saf…

This phase 2 trial tests a therapy called AlloNK, made from donor cord blood immune cells, combined with the drug rituximab. It aims to see if this combination is safe and can help people with severe forms of rheumatoid arthritis, Sjögren's disease, myositis, or scleroderma that …

This Phase 2 trial tests an experimental drug called INCB000928 (zilurgisertib) in people with fibrodysplasia ossificans progressiva (FOP), a rare genetic condition where soft tissues turn into bone. About 98 participants aged 2 and older will receive either the drug or a placebo…

This early-stage study tests a new drug called SAR448501 (DR-0201) in 62 adults with lupus or rheumatoid arthritis. The drug is a bispecific antibody designed to target the immune system. The main goal is to check safety and find the right dose, not yet to prove it works. Partici…

This study tests whether giving patients electrical muscle stimulation and high-protein shakes soon after a type of stroke (aneurysmal subarachnoid hemorrhage) can prevent muscle loss and improve recovery. Thirty adults will receive the combo therapy or standard care. The main go…

This Phase 2 study tests whether a Traditional Chinese Medicine (TCM) herbal formula can improve muscle strength and bone health in pre-frail adults aged 65 and older, compared to the nutritional supplement HMB. Over 24 weeks, 90 participants will take either the TCM powder or HM…

This study tests an investigational drug called ENTR-601-45 in people with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 45 skipping). The goal is to check the drug's safety, find the right dose, and see if it can help muscles work better. About 24 ma…

This study is testing whether a drug called tadalafil (often used for erectile dysfunction) combined with a home cycling program can help boys with Duchenne muscular dystrophy. The drug aims to improve blood flow to muscles, which is often poor in DMD, while exercise builds stren…

This study tests the long-term safety and effectiveness of dazukibart in people with dermatomyositis or polymyositis, rare diseases that cause muscle weakness and rashes. Participants who completed a previous study can join. Some will receive the drug via IV every 4 weeks for abo…

This study tests a new medicine, DYNE-101, in 116 adults with myotonic dystrophy type 1 (DM1), a condition that causes muscle weakness and stiffness. The main goals are to check if the drug is safe and if it can reduce muscle stiffness and improve muscle function. Participants re…

This phase 2 trial is testing whether filgotinib, a drug already used for arthritis, can help people with three rare immune diseases: Behçet's disease, myositis, and IgG4-related disease. The goal is to see if it can control disease activity and reduce the need for long-term ster…

This early-phase trial is testing a new cell therapy called QH103 for people with severe autoimmune diseases like multiple sclerosis and myasthenia gravis that haven't responded to standard treatments. The therapy uses specially engineered immune cells to target and attack the fa…

This early-phase trial tests a gene therapy called SPOT-03 in 9 boys with Duchenne muscular dystrophy (DMD), aged 2 to 8. The main goal is to see if the treatment is safe and tolerable. Researchers will also check if it increases dystrophin protein levels in muscles, which could …

This early-stage study tests a new treatment called HBI0101 CAR T for people with severe autoimmune diseases like lupus, multiple sclerosis, and rheumatoid arthritis that haven't improved with standard treatments. The main goal is to find a safe dose and check for side effects in…

This study is testing a drug called deucravacitinib in 10 adults with idiopathic inflammatory myopathies, a group of rare diseases that cause muscle inflammation and weakness. Participants will take one pill daily for 6 months. Researchers will track symptom changes and lab resul…

This large real-world study is testing whether platelet-rich plasma (PRP) injections can reduce pain and improve function in people with chronic tendinopathies, including patellar, Achilles, elbow, and plantar fasciitis. Researchers will follow 2000 participants for 12 months, tr…

This study tests a new treatment called anti-CD19/BCMA universal CAR-T cells for people with severe autoimmune diseases like rheumatoid arthritis, lupus, and scleroderma. The therapy aims to reset the immune system by targeting and removing faulty B cells. About 72 adults will re…

This study tests whether stem cells from umbilical cord lining can safely reduce symptoms of dermatomyositis and polymyositis, two autoimmune diseases that cause muscle weakness and inflammation. About 40 adults will receive three doses of stem cells or a placebo, then switch. Re…

This study tests an investigational drug called PGN-EDODM1 in 24 adults with myotonic dystrophy type 1, a condition causing muscle stiffness and weakness. Participants receive multiple doses of the drug or a placebo by IV to check safety and how the body processes it. The goal is…

This Phase 2 trial tests a personalized cell therapy called Descartes-08 for people with dermatomyositis or antisynthetase syndrome, two rare autoimmune diseases that cause muscle weakness and inflammation. The therapy uses a patient's own immune cells, modified to target and red…

This early-phase trial is testing EPI-321, a one-time gene therapy designed to silence the faulty gene that causes facioscapulohumeral muscular dystrophy (FSHD). The study will enroll 12 adults with FSHD Type 1 to see if the treatment is safe and tolerable, and whether it shows a…

This early-stage trial tests a new biologic drug called ARGX-119 in 16 adults with a rare genetic muscle weakness condition called DOK7-congenital myasthenic syndrome (CMS). The study aims to see if the drug is safe and how the body processes it. Participants will receive either …

This early study is testing a new treatment called CD19 CAR-T therapy for people with severe autoimmune diseases like lupus and scleroderma that haven't improved with standard medications. The therapy involves taking a patient's own immune cells, reprogramming them to target and …

This study tests a one-time gene therapy called RGX-314 for wet age-related macular degeneration (AMD), a leading cause of vision loss. About 714 adults aged 50-89 who currently need regular eye injections will receive either the gene therapy or standard treatment. The goal is to…

This early-stage trial is testing a new biologic drug called CC312 in just 6 adults with lupus, myositis, or scleroderma that has not responded to standard treatments. The main goal is to check safety and find the right dose, not yet to prove it works. Participants receive multip…

This phase 2 trial tests whether a 4-week course of an essential amino acid supplement can help older adults (65+) rebuild muscle after a hip fracture. Sixty participants will be randomly assigned to receive either the supplement or standard care alone. Researchers will measure m…

This early-stage study tests an injection called GT719 in 30 people with moderate to severe autoimmune diseases like lupus that haven't responded to standard treatments. The main goal is to check safety and find the right dose, while also looking at whether it helps control disea…

This study is testing a new medical device called the lok™ suture anchor system for repairing torn tendons and labrum in the shoulder. About 216 adults with rotator cuff tears, shoulder instability, or partial tendon avulsions will receive the anchors during surgery. Researchers …

This program provides expanded access to triheptanoin for people with long-chain fatty acid oxidation disorders (LC-FAOD) who have few treatment options and cannot join a clinical trial. The goal is to help manage the disease by providing an alternative energy source for the body…

This study tests a brain implant that records and stimulates brain areas to help adults with severe paralysis (from spinal cord injury, ALS, or stroke) control assistive devices like computers or wheelchairs. The main goal is to check safety, and researchers will also see if part…

This early-stage trial is testing Descartes-08, a cell therapy made from modified immune cells, in 50 young people aged 12 and older with four autoimmune diseases: childhood-onset lupus, ANCA-associated vasculitis, juvenile myasthenia gravis, and juvenile dermatomyositis. The stu…

This early-stage trial is testing a drug called RAY121 in 144 people with one of six immune diseases, including antiphospholipid syndrome and bullous pemphigoid. The drug works by blocking a specific part of the immune system (the complement pathway) that can cause tissue damage.…

This study is testing the long-term safety of an experimental drug called PGN-EDODM1 in 48 adults with myotonic dystrophy type 1 (DM1) who have already taken the drug in a previous study. Participants will receive the drug by IV infusion and be monitored for side effects. The goa…

This early-phase trial is testing a new treatment called KN5601, which uses a patient's own immune cells (NK cells) that have been engineered to target and destroy harmful B cells. The goal is to see if it is safe and can help children aged 5 and older with rheumatic diseases lik…

This study tests an investigational drug called ENTR-601-44 in 24 boys and young men with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping a specific part of the gene (exon 44). The trial has two parts: first, to find the safest and most effecti…

This early study tests the safety of a new biologic drug called STR-P004 in 39 adults with autoimmune diseases that have not responded to standard treatments. Participants receive the drug by IV infusion, and researchers monitor for side effects and how the drug behaves in the bo…

This study tests an experimental drug called NS-089/NCNP-02 (Brogidirsen) in 20 boys with Duchenne muscular dystrophy whose genetic mutation can be fixed by skipping exon 44. The drug is given as a weekly IV infusion and aims to help the body produce a working version of the dyst…

This early-phase trial is testing a personalized cell therapy called CAR-T, designed to target and destroy faulty immune cells in people with active autoimmune diseases like lupus, scleroderma, and Sjogren's syndrome. Up to 20 participants will receive the treatment after a short…

This early-stage study tests a new cell therapy called YTS109 in 18 adults aged 18-65 with autoimmune diseases like lupus and Sjogren's syndrome that have not responded to standard treatments. Participants receive a single infusion of the cells, and researchers monitor safety and…

This study tests a gene therapy designed to help older adults regain muscle mass and strength lost with age. The treatment uses a harmless virus to deliver a gene that promotes muscle growth, sometimes combined with another gene that improves blood flow. The trial involves 12 par…

This early-phase trial tests whether injecting lab-grown muscle cells (MyoPAXon) into the foot is safe for adults with Duchenne muscular dystrophy who can no longer walk. Eight participants will receive the cells along with an immunosuppressant drug to prevent rejection. The main…

This phase 3 trial tests whether belimumab (Benlysta) can stabilize or improve lung function in adults with interstitial lung disease caused by connective tissue diseases like rheumatoid arthritis or lupus. About 440 participants will receive either belimumab injections or a plac…

This study looks at whether anti-inflammatory drugs (like anakinra or canakinumab) can reduce painful flare-ups and prevent extra bone growth in people with a rare genetic condition called fibrodysplasia ossificans progressiva (FOP). Eleven people with severe FOP will be tracked …

This early-stage trial is testing a new treatment called YTS109 for people with severe autoimmune diseases like lupus and Sjogren's syndrome that have come back or not responded to standard care. About 18 adults will receive a single infusion of these specially engineered immune …

This study tests whether giving ICU patients extra protein and activating their muscles early (using electrical stimulation and movement) can prevent severe muscle loss. About 40 critically ill adults on ventilators will take part. The goal is to see if this approach helps patien…

This early-stage study tests a new cell therapy called YTS109 in 18 adults with severe autoimmune diseases like lupus and Sjogren's syndrome that have not responded to standard treatments. Participants receive a single infusion of the cells, and researchers monitor safety and how…

This early-stage study tests a new cell treatment called YTS109 in 18 adults with hard-to-treat autoimmune diseases like lupus and scleroderma. Participants receive a single infusion of these specially designed cells. The main goal is to check if the treatment is safe, and also t…

This study follows 250 boys (ages 2 and older) with Duchenne muscular dystrophy who are taking vamorolone (AGAMREE). Researchers will monitor side effects, growth, bone health, heart function, and quality of life over time. The goal is to better understand the long-term safety an…

This phase 2 trial tests whether intravenous immunoglobulin (IVIG) can help people with a rare autoimmune muscle disease called anti-HMGCR immune-mediated necrotizing myopathy. Twelve participants will receive either IVIG or a placebo every 4 weeks for 12 weeks, then all may rece…

This early-stage study is testing whether clenbuterol, a drug used for breathing problems, is safe for people with facioscapulohumeral muscular dystrophy (FSHD). About 30 adults with FSHD will receive one of three doses of the drug. Researchers will monitor side effects, heart ra…

This study tests a single dose of a gene therapy called AB-1003 in 10 adults with a rare genetic muscle disease (LGMD2I/R9). The goal is to see if it is safe and can help improve muscle function. Participants must be able to walk or run 10 meters in under 30 seconds.

This study tests whether a drug called ACM-001.1 can help obese people keep their muscle while taking semaglutide for weight loss, and help them regain muscle after stopping semaglutide. About 120 participants will receive either ACM-001.1 or a placebo alongside semaglutide, and …

This early-phase study tests a new treatment called TI-0032-III for people with severe autoimmune diseases like lupus and scleroderma that haven't improved with standard care. The treatment uses a special injection to create CAR-T cells inside the body, which then target and dest…

This study tests a new drug called ATX-01 in 56 adults with myotonic dystrophy type 1, a condition that causes muscle stiffness and weakness. The drug aims to block a molecule that may contribute to the disease. The trial will first give a single dose, then multiple doses, to che…

This study tests an experimental drug called ARO-DUX4 in people with facioscapulohumeral muscular dystrophy type 1 (FSHD1), a genetic condition that causes muscle weakness. The main goal is to check the drug's safety and how the body processes it. About 60 adults and teens will r…

This early-phase study tests a new treatment called RD06-05, which uses a patient's own immune cells modified to target and reset the immune system in autoimmune diseases like lupus, multiple sclerosis, and vasculitis. The goal is to check safety and how well the body tolerates i…

This early-stage trial is testing a new treatment called LUCAR-G79D for people with lupus or inflammatory myopathies that haven't responded to standard therapies. The treatment involves giving patients specially engineered immune cells to help control their disease. The study is …

This study tests whether tocilizumab, a drug that blocks inflammation, can improve symptoms and breathing in adults with anti-MDA5+ dermatomyositis, a rare autoimmune disease affecting muscles, skin, and lungs. About 110 participants will receive either tocilizumab or a placebo e…

This phase 1 trial tests N-acetylcysteine (NAC), an antioxidant, in 18 adults with a specific mitochondrial disease caused by the m.3243A>G mutation and low brain glutathione levels. Participants take one of three daily doses (1800, 3600, or 5400 mg) for 3 months to find the safe…

This early-stage study tests a new treatment called RXIM002 for people with severe autoimmune diseases that haven't responded to standard therapies. The treatment uses a special RNA molecule to create immune cells that target and destroy faulty B cells, which are causing the dise…

This program offers ongoing access to vamorolone, a steroid-like drug, for boys with Duchenne muscular dystrophy who finished earlier studies. It aims to keep them on treatment while the drug is being reviewed for approval. Participants take vamorolone daily by mouth at doses cho…

This study looks at the long-term safety and effects of the drug givinostat in people with Duchenne muscular dystrophy (DMD). It is open to those who have already taken part in a previous givinostat study. The goal is to track side effects and see how well the drug works over tim…

This early-stage trial tests a new treatment for people with autoimmune diseases like lupus and multiple sclerosis that haven't responded to standard therapies. The treatment uses a patient's own immune cells, modified in a lab to target and destroy the faulty cells causing the d…

This early-stage study is testing a new type of cell therapy (called QT-019C) in 12 adults with severe autoimmune diseases like lupus and scleroderma that have not responded to standard treatments. The therapy uses specially engineered immune cells from a healthy donor to target …

This study follows 175 people with spinal muscular atrophy who previously received the gene therapy Zolgensma in clinical trials. Researchers will monitor them for 5 years to track serious side effects and developmental milestones. The goal is to understand how safe and effective…

This early-phase study tests whether a combination of two drugs, pozelimab and cemdisiran, can improve physical function in people with sporadic inclusion body myositis (sIBM), a rare muscle disease that causes progressive weakness. Ten adults aged 45 to 75 with sIBM will receive…

This early-phase study tests a single injection of a gene therapy called V001-BCMA for people with autoimmune diseases like lupus that have not responded to standard treatments. The therapy targets and modifies specific immune cells to reduce disease activity. The main goal is to…

This early-phase trial tests a new treatment called GC012F, which uses a patient's own immune cells (CAR-T cells) to target and attack the cause of idiopathic inflammatory myopathy, a group of rare muscle diseases. The study will enroll 12 adults whose disease has not responded t…

This study tests a single injection of BB-301 directly into throat muscles to improve swallowing in people with oculopharyngeal muscular dystrophy (OPMD). About 30 adults aged 50-65 with swallowing problems will receive the injection during a short surgery. Researchers will check…

This study tests a new treatment using specially engineered immune cells (CAR-NK) from cord blood for people with severe autoimmune diseases like lupus, scleroderma, and vasculitis that haven't responded to standard therapies. The goal is to see if it's safe and can control the d…

This early-phase trial is testing a personalized immune cell therapy called CD19 CAR-T in 15 people with severe autoimmune diseases like lupus and scleroderma that haven't responded to standard treatments. The therapy aims to temporarily deplete certain immune cells to calm the o…

This study tests whether taking whey protein and collagen supplements along with doing resistance exercises can help people keep their muscle mass after bariatric surgery. About 35 adults who are having the surgery will be split into groups to compare different combinations of su…

This study tests an experimental drug called WVE-N531 in 26 people with Duchenne muscular dystrophy who have a specific genetic flaw (exon 53 mutation). The drug is designed to help the body make a shortened but working version of dystrophin, a protein missing in DMD. The trial w…

This early-phase study is testing a new cell therapy called LUCAR-DKS1 in 36 adults with relapsed or refractory autoimmune diseases like lupus. Participants receive their own modified NK cells after a short chemotherapy prep. The main goals are to check safety, find the right dos…

This early-stage study tests whether metformin, a widely used diabetes medication, can improve muscle regrowth in older adults after a period of inactivity (like bed rest). Fifty volunteers aged 60 and older will receive either metformin or a placebo during recovery. The goal is …

This early-stage study tests a gene therapy called BBM-D101 in 9 boys aged 4 to 8 with Duchenne muscular dystrophy (DMD). The goal is to see if it is safe and can help produce a missing protein in muscles. Researchers will monitor side effects and measure changes in muscle health…

This study looks at whether adding protein supplements to the diet can prevent muscle wasting in people with heart failure. About 120 adults with heart failure and reduced pumping ability will be randomly assigned to receive no, low, or high doses of protein drinks. The main goal…

This study tests a new drug called ARO-DM1 in 78 adults aged 18-65 with type 1 myotonic dystrophy, a genetic muscle disorder. The goal is to check safety and how the body processes the drug. Participants receive either ARO-DM1 or a placebo by IV or injection. It is an early-stage…

This early-phase trial tests a new drug called IMM01-STEM, made from stem cell secretions, in 28 people with muscle wasting due to knee osteoarthritis. The main goal is to check safety and find the right dose. Participants receive injections and are monitored for side effects ove…

This study tests a wearable robotic exoskeleton designed to improve walking in children aged 3 to 17 with conditions like cerebral palsy, spina bifida, muscular dystrophy, or incomplete spinal cord injury. The device fits on the legs and can assist or resist movement during walki…

This study tests a cream containing ruxolitinib on the skin of 15 adults with dermatomyositis whose skin symptoms haven't improved with standard treatments. The cream is applied to affected areas for up to 12 weeks to see if it reduces redness, scaling, and other signs of disease…

This trial tests a one-time treatment using a patient's own immune cells, engineered to target and destroy B cells, which drive autoimmune diseases like lupus, scleroderma, and myositis. 24 adults with active disease will receive the therapy after a short chemotherapy. The main g…

This early-stage study tests a new treatment called RD06-05, a universal CAR-T cell therapy, in up to 84 adults with autoimmune diseases like lupus, vasculitis, and scleroderma. The therapy targets and removes faulty immune cells to help control the disease. The main goal is to c…

This early-stage study tests a new treatment for people with severe autoimmune diseases like lupus and scleroderma that haven't improved with standard therapies. The treatment uses specially engineered immune cells (CAR T-cells) from a healthy donor to target and potentially rese…

This early-stage trial tests a treatment called CAR-T cell therapy for people with severe autoimmune diseases that haven't responded to other treatments. The therapy uses a patient's own immune cells, modified to target and reset the immune system. Only 3 participants will be enr…

This early-phase trial is testing an injection called GT719 in 10 adults with moderate to severe autoimmune diseases that haven't responded to other treatments. The main goal is to check safety and find the right dose. Researchers will also look for signs that the therapy might h…

This early-stage trial tests a new type of CAR-T cell therapy in 11 children with severe rheumatic diseases like juvenile dermatomyositis and arthritis that haven't improved with standard treatments. The therapy uses specially engineered immune cells to target and calm the overac…

This early-phase study is testing a new drug called CC312 in 18 adults with severe autoimmune diseases like lupus and rheumatoid arthritis that haven't improved with standard treatments. CC312 is a type of immune therapy designed to target and destroy faulty B cells, similar to C…

This early-phase study tests a new cell therapy called BZE2204 in 20 people with lupus, myositis, or immune thrombocytopenia (ITP) that has not responded to standard treatments. The therapy uses a patient's own immune cells, modified to target and calm the overactive immune syste…

This study tests an oral drug called TTI-0102 in 12 people with MELAS, a rare genetic disorder that causes muscle weakness, strokes, and fatigue. Participants receive either the drug or a placebo for 6 months. Researchers will measure walking ability, fatigue, and quality of life…

This study tests an experimental drug called SCT520FF for wet age-related macular degeneration (AMD), a leading cause of vision loss. About 82 adults aged 45 to 80 will receive either SCT520FF or an existing treatment (EYLEA) via eye injections. Researchers will check safety, sid…

This study aims to see if a special MRI designed for teeth can diagnose jaw joint disorders, tooth infections, and help plan dental implants as well as or better than traditional X-rays. About 470 adults needing dental imaging will get both an MRI and standard X-rays, and the res…

This study is testing a new blood test that can diagnose single-gene disorders in unborn babies using a sample from the mother. The test looks at fetal DNA found in the mother's blood, which is safer than traditional invasive methods that carry a small risk of miscarriage. Resear…

This study is testing a handheld device called mScan that uses a tiny, painless electrical current to measure muscle health. Researchers want to see if it can give similar results to an MRI, but faster and more conveniently. The study involves 150 adults with and without muscle d…

This study is testing a small device called a nanosensor that measures oxygen levels in muscle tissue. The goal is to see if it can accurately assess mitochondrial function in people with mitochondrial myopathy compared to healthy volunteers. If it works, this sensor could become…

This early-stage study tests a small nanosensor placed under the skin in the forearm to measure oxygen levels in muscle, which reflects how well mitochondria are working. Researchers will compare results from 24 people—some with mitochondrial myopathy and some healthy—to see if t…

This study aims to create a new scoring system called HASS to reliably diagnose long biceps tendon injuries before surgery. Currently, these injuries are hard to confirm without surgery, leading to uncertainty. Researchers will combine physical exam tests and imaging data from 20…

This study tests a special MRI technique to see if it can tell the difference between normal and painful jaw muscles in people with masticatory myofascial pain syndrome. Researchers will scan the jaw muscles of 90 adults and compare the results. The goal is to find a better way t…

This study is testing a new device that uses near-infrared light to measure pelvic floor muscle fitness in women with myofascial pelvic pain, a condition that affects up to 26% of women. The goal is to create a simple, non-invasive diagnostic tool that could replace the need for …

This study is testing a new blood-based test that aims to predict which medication will work best for each person with rheumatic diseases like rheumatoid arthritis or lupus. Researchers will take a blood sample and analyze immune cells to create a personalized treatment profile. …

This study tests whether wearing a smartwatch that tracks activity and body composition, along with exercise reminders, can help people on GLP-1 drugs (like Ozempic or Wegovy) maintain muscle and strength. About 100 adults who recently started or plan to start these medications w…

This study is testing a gel called OSTENIL TENDON to see if it safely reduces pain and improves movement in people with tendon problems. Researchers will enroll 84 adults with one of four types of tendinopathy. The main goal is to measure pain changes three months after treatment…

This study tests different dry needling and electroacupuncture methods on healthy adults with hidden muscle knots in the forearm. 150 participants will be split into five groups to see which technique causes the least soreness and best improves grip strength and movement. Results…

This study tests whether the drug sonlicromanol can reduce fatigue and improve physical abilities like balance and leg strength in adults with a specific genetic form of mitochondrial disease. About 220 participants will take either the drug or a placebo twice daily for 52 weeks.…

This study tests whether a personalized mix of endurance and strength exercises, done at home with video check-ins, can improve mobility and muscle strength in people with mitochondrial myopathy. Fifteen adults with confirmed genetic mutations will follow the program for up to 12…

This study tests whether a gentle brain stimulation technique called tDCS, applied before eccentric knee exercises, can improve pain and function more than exercise alone in people with patellar tendinopathy (jumper's knee). Forty-two adults with chronic knee pain will be randoml…

This study tests whether adding hyaluronic acid to a standard corticosteroid injection works better for shoulder tendonitis pain than the steroid alone. About 132 adults with confirmed supraspinatus tendinopathy will receive either the combo or a placebo injection. Pain levels ar…

This study tests whether a numbing injection called Twin Block can reduce jaw muscle pain and the need for opioid painkillers after wisdom tooth removal. About 48 healthy adults who have jaw pain the day after surgery will receive either the Twin Block or a placebo. Researchers w…

This early-stage study is testing whether using a special muscle-mapping device to guide Botox injections can better relieve chronic pelvic floor pain in women compared to the standard injection method. Researchers will enroll 30 women with long-term pelvic pain and give them two…

This study tests whether a personalized exercise program can improve balance and physical function in adults with rare neuromuscular disorders like Charcot-Marie-Tooth disease, facioscapulohumeral muscular dystrophy, and myotonic dystrophy type 1. Participants will receive a 12-d…

This study tests a short, two-session telehealth program called Physical Self-Regulation for people with chronic masticatory muscle pain (jaw pain). Participants will be randomly assigned to either this program or a control session that teaches about jaw anatomy. The goal is to s…

This study tests three types of injections for people with chronic neck and shoulder muscle pain caused by trigger points. It compares platelet-rich plasma (PRP), a mix of PRP and steroid, and steroid alone to see which works best. 150 adults aged 18-65 with a single active trigg…

This study tests whether taking omega-3 fish oil capsules and doing simple resistance exercises with elastic bands can help cancer patients maintain muscle mass and strength. It involves 288 adults with various cancers who are at risk of muscle wasting. The goal is to improve phy…

This study compares two common injection treatments for chronic mid-back pain: the erector spinae plane (ESP) block and trigger point injection (TPI). About 76 adults with pain lasting at least three months will be randomly assigned to one treatment. Researchers will track pain l…

This study tests whether playing virtual reality games can help people with Duchenne muscular dystrophy improve their arm and hand function. 36 participants will either receive standard physiotherapy or the same therapy plus VR games using a Meta Quest 3 headset. The games are de…

This study tests whether a special laser device (Erchonia EVRL) can temporarily ease chronic jaw pain caused by TMJ disorder. Researchers will compare the laser to a placebo in 64 adults who have had constant jaw pain for at least 3 months. The main goal is to see if more people …

This study looks at whether doing breathing exercises before orthopedic surgery (like knee, hip, or fracture repair) can help your lungs work better after the operation. It involves 36 adults who have used tobacco and are at risk for breathing problems after surgery. Participants…

This study tests a home monitoring program called LiverWatch for people with advanced liver disease (cirrhosis). Participants get a Fitbit, personalized nutrition advice, and weekly check-ins to boost activity and manage symptoms. The goal is to see if this approach improves qual…

This study tests a powered leg brace called the Nomad in 36 people who have trouble walking due to conditions like stroke, multiple sclerosis, or spinal cord injury. Participants will use the brace at home for three months to see if it improves their walking speed, balance, and q…

This study compares two electrical frequencies (high vs. low) used in percutaneous electrical nerve stimulation (PENS) for shoulder pain. Thirty adults with a trigger point in the infraspinatus muscle will receive a single session, and researchers will measure changes in muscle s…

This study compares three common treatments for long-lasting muscle pain: dry needling, shock wave therapy, and pulsed radiofrequency. Researchers will enroll 60 adults with myofascial pain syndrome who have not improved with standard care. The goal is to find which method reduce…

This study looks at whether a custom mouthguard (occlusal splint) can reduce pain and improve muscle function in people with teeth grinding (bruxism) and related jaw and neck muscle pain. About 40 adults will be split into two groups: one gets the mouthguard right away, the other…

This study tests whether injecting sodium thiosulfate directly into painful calcium deposits (calcinosis) can shrink them and ease symptoms. Twenty adults with scleroderma, dermatomyositis, or mixed connective tissue disease will receive ultrasound-guided injections. The main goa…

This study looks at 134 people with De Quervain's disease, a painful thumb tendon condition. All participants get a steroid injection, then either standard physiotherapy or a program of eccentric exercises. The goal is to see which approach better reduces pain and improves hand f…

This study looks at whether adding a simple nerve-stimulation technique (using deep breathing to activate the vagus nerve) to standard physical therapy can better reduce pain and improve jaw movement in people with jaw joint disorders. Sixty adults with jaw muscle pain will recei…

This study tests whether using Doppler ultrasound to detect inflammation around calcium deposits in the shoulder can improve the results of a guided injection. 100 adults with chronic shoulder pain and calcific tendinitis will receive an ultrasound-guided injection, with or witho…

This study tests whether a small device that gently stimulates a nerve in the ear can help people with long-term back pain. 48 adults will either get the ear stimulation plus standard rehab, or rehab alone, for 3 weeks. The goal is to see if the device improves daily function and…

This study tests a handheld device called Sonodyn for people with chronic back, neck, or tension headache pain that hasn't improved with other treatments. The device uses a mix of low-energy ultrasound, electrical, and magnetic pulses to target painful trigger points. About 126 a…

This study tests whether combining electroacupuncture with vacuum cupping therapy works better than electroacupuncture alone for shoulder tendinitis. Fifty-four adults with shoulder pain and stiffness will receive either electroacupuncture alone or electroacupuncture plus cupping…

This study tests whether taking amino acid supplements, along with a special diet and exercise program, can help people with facioscapulohumeral muscular dystrophy (FSHD) gain muscle, lose fat, and improve physical abilities like walking and strength. About 48 adults with FSHD an…

This study tests whether a physical therapist-guided exercise program can improve strength and walking ability in adults with myotonic dystrophy type 2 (DM2). Twenty-four participants will be split into three groups, each doing the same exercises but with different levels of ther…

This study tests whether injecting platelet-rich plasma (PRP) into acupuncture points can reduce chronic neck pain caused by muscle strain. 72 adults with neck pain lasting over 3 months will receive either PRP or a placebo (salt water) injection. Researchers will measure pain le…

This study looks at whether one session of dry needling can quickly reduce calf muscle pain and improve sprint speed and jumping ability in amateur football players aged 18 to 25. Sixty participants will receive either real or sham needling, and their pain, speed, and performance…

This study tests whether a device that delivers gentle electrical stimulation to the pelvic floor can reduce chronic pelvic pain in women. Sixty women with myofascial pelvic pain will either receive the device treatment or standard care (education and exercises). The goal is to s…

This study tests whether focused shock wave therapy can reduce pain and improve jaw function in people with temporomandibular disorders (TMD) of muscular origin. One hundred adults with moderate to severe jaw pain for at least 3 months will receive either shock wave therapy or a …

This study compares three treatments—exercise, dry needling, and a newer injection called interfascial block—for people with long-lasting pain in the upper back muscle (trapezius). About 78 adults aged 18 to 55 with at least 3 months of pain will be assigned to one of the three g…

This study is testing whether a device called TENS, which sends mild electrical pulses through the skin, can safely reduce chronic low back pain. Forty adults with mild pain will use the device for 20-30 minutes, five times a week for four weeks. Researchers will track pain level…

This study tests whether whole-body electrical muscle stimulation (WB-EMS) can help adults with neuromuscular diseases like ALS, SMA, and muscular dystrophy exercise safely. Because these conditions weaken the nerves that control muscles, traditional exercise is often too hard. W…

This study tests whether online or group physiotherapy can be as effective as individual in-person sessions for treating jaw muscle pain (TMD). About 90 adults with jaw muscle pain will be randomly assigned to one of three treatment formats. The goal is to find more accessible wa…

This study compares a new injection of liquid phase concentrated growth factor (LPCGF) to standard arthrocentesis (joint washing) for people with painful jaw clicking and limited mouth opening due to a displaced disc. Thirty-two adults aged 18-45 with unilateral disc displacement…

This study tests different physical therapy combinations for people with shoulder tendon pain (supraspinatus tendinitis). Researchers will measure pain, range of motion, and tendon thickness using ultrasound. 60 adults aged 40-60 with shoulder pain for up to 3 months will be assi…

This study looks at whether a quick session of electrical stimulation can lower pain and help people with Achilles tendon injuries move better. About 30 adults with Achilles tendinopathy or a past rupture will try two different stimulation methods. The goal is to see if this appr…

This study compares two exercise programs for people with long-term pain in the Achilles, patellar tendon, or plantar fascia. One group does standard strength training, while the other adds early jumping (plyometric) exercises. Researchers will measure jump function after 12 week…

This study compares a high-tech rehabilitation program—using robotic systems, virtual reality, and balance platforms—to standard physical therapy for people with acute neuromuscular diseases like Guillain-Barré syndrome. About 124 adults aged 18 to 80 will participate. The goal i…

This study tests whether a program of exercise and ergonomics training can reduce muscle and joint pain in teachers. About 80 teachers who have had pain for at least three months will be randomly assigned to receive the training or not. Researchers will measure changes in pain, d…

This study looks at whether the Alexander technique, a method that teaches better posture and movement habits, can help children aged 5 to 9 with Duchenne muscular dystrophy control their upper limbs and feel better day-to-day. About 33 kids will take part, and researchers will m…

This study looks at whether dry needling can reduce pain from trigger points (tight, tender spots) in the trapezius muscle, which runs from the neck to the shoulders. About 70 adults with myofascial pain will receive either dry needling or a placebo treatment. Researchers will us…

This study tests whether a daily 3-month breathing exercise program can improve strength and daily function in people with inflammatory myopathy, a rare autoimmune disease that causes muscle weakness. Thirty-three adults will use a handheld device at home to strengthen their brea…

This study explores whether weekly music and movement sessions are practical and enjoyable for children aged 6 to 18 with myotonic dystrophy type 1 (DM1). Over 10 weeks, participants attend 45-minute music classes, undergo physical and cognitive tests, and provide biological samp…

This study tests whether adding fascial manipulation to standard shock wave therapy improves pain and knee function in people with patellar tendinitis (jumper's knee). 38 adults aged 18-45 will be randomly assigned to receive either shock wave therapy alone or combined with targe…

This study tests a web-based app called IAMABLE that provides evidence-based rehabilitation strategies for people with chronic conditions like heart disease, arthritis, or neurological disorders. Fifty adults aged 45 to 75 will use the app for 4 months to set goals and learn abou…

This study looks at whether adding magnesium sulfate to a standard lidocaine injection can better relieve pain and improve muscle function in people with myofascial pain dysfunction syndrome (chronic jaw muscle pain). About 28 adults with active trigger points will receive either…

This Phase 3 trial tests whether a once-daily dose of mexiletine PR can safely reduce muscle stiffness (myotonia) in people with myotonic dystrophy types 1 and 2. About 176 participants will receive either the drug or a placebo for 26 weeks. The main measure is how quickly hand m…

This study tests a minimally invasive procedure called transcatheter arterial embolization (TAE) combined with physical therapy for people with chronic shoulder pain from rotator cuff tendinopathy that hasn't improved with standard treatments. About 41 adults with moderate to sev…

This study tests whether a fun, game-based occupational therapy program can improve quality of life, daily skills, and emotional health in 20 teens with Duchenne muscular dystrophy. Participants will use a computer or tablet at home. Researchers will compare results to standard t…

This study looks at two different exercise programs—using flexi bands or whole-body electrical stimulation—for people with cancer. The goal is to see which one better helps maintain muscle strength, reduce inflammation, and improve quality of life during treatment. About 100 part…

This study tests two noninvasive treatments for jaw muscle pain (TMD): a standard mouth splint and a newer ear stent. About 32 adults with moderate to severe pain will be randomly assigned to one treatment. Researchers will measure changes in muscle activity, mouth opening, and p…

This study compares two types of injections for people with chronic migraines or headaches. One targets trigger points in muscles, the other blocks a nerve in the neck. The goal is to see if the simpler trigger point injection works just as well. About 1,000 adults aged 18-65 wil…

This study tests whether injecting a common steroid into muscle trigger points can relieve long-term shoulder pain and certain headaches as well as injecting into the joint. About 1,000 adults aged 45 and older with shoulder arthritis will receive either a trigger point or joint …

This study tests whether injecting a steroid and numbing medicine into muscle trigger points can relieve knee osteoarthritis pain as well as injecting into the knee joint itself. About 1,000 adults with knee arthritis and at least one tender muscle spot will receive either a trig…

This study tests whether injecting a steroid and numbing medicine into muscle trigger points can relieve chronic cervicogenic headaches (headaches that start from the neck). Over 1,000 adults aged 18-65 with at least 3 months of headaches will receive either the trigger point inj…

This study compares two types of injections for hip osteoarthritis pain: one into the joint and one into nearby muscle trigger points. Over 1,000 adults with hip pain will be randomly assigned to one of the two groups and followed for two years. The goal is to see if the simpler …

This study compares two common physical therapy techniques—Mulligan and Maitland mobilization—for treating temporomandibular joint (TMJ) dysfunction, which causes jaw pain and limited movement. Thirty adults with TMJ pain will be randomly assigned to one of the two techniques, co…

This study tests whether adding dry needling to standard physical therapy helps people with jaw pain (TMJ dysfunction). One hundred adults will either get standard rehab alone or rehab plus dry needling. Researchers will measure pain, jaw movement, and quality of life to see if t…

This study looks at whether adding blood flow restriction training (BFRT) to standard physical therapy helps teens recover strength and muscle size after ACL reconstruction. Forty teens who played sports before surgery will be randomly assigned to either standard rehab or standar…

This study tests a new home-based program that uses a special treadmill and virtual reality to help children with conditions like cerebral palsy, muscle diseases, or obesity improve their walking. About 30 children aged 6 to 17 will try the program at home for at least 3 sessions…

This study compares two different doses of a treatment called percutaneous electrolysis for people with chronic neck pain caused by muscle knots in the upper trapezius. Forty adults will receive either a low-intensity or high-intensity version of the treatment, along with stretch…

This early-stage study tests whether stem cells from umbilical cord lining can safely help people with FSHD, a genetic muscle-weakening disease. Sixteen adults will receive two doses of the cells and two doses of a placebo (saline) through an IV, in random order. The main goal is…

This study looks at how different types of electrical nerve stimulation affect leg strength in athletes with patellar tendinopathy (jumper's knee). Nineteen athletes will try three stimulation methods in separate sessions to see which one best improves quadriceps strength. The go…

This study tests a special fabric shoulder brace designed to mimic muscle support for people with neuromuscular disorders like muscular dystrophy, SMA, and ALS. About 30 participants will wear the brace and perform arm tasks to see if it improves movement, strength, and daily fun…

This phase 3 trial is testing whether metformin, a common diabetes drug, can improve muscle function in adults with myotonic dystrophy type 1 (Steinert's disease). The study will enroll 142 participants who will receive either metformin or a placebo for 6 months. Researchers will…

This study tests whether a special laser (LightForce) can reduce shoulder pain caused by inflammation from conditions like impingement or rotator cuff tendinitis. About 130 adults with pain lasting over 3 months will get either real laser or a sham laser, plus standard physiother…

This study tests whether extra support for case managers helps veterans leaving the military better manage pain and reduce risky substance use. About 1800 veterans will take part. The program focuses on non-drug pain treatments and counseling.

This study tests whether a stabilizing occlusal splint (a type of mouthguard) can reduce tinnitus (ringing in the ears) in people who also have temporomandibular joint (TMJ) disorder. Researchers will compare the real splint to a placebo splint in 63 adults over 4 months, measuri…

This study follows up to 10 children from birth to under 6 years old who have genetic muscle disorders causing stiffness (myotonia) and are taking mexiletine. Researchers will collect data on side effects and how the medicine is used in routine care over the long term. The goal i…

This study tests two different exercise programs—Muscle Energy Technique and Neck Calliet Exercises—to see which better reduces neck pain and disability in nurses with trigger points. Thirty-eight female nurses will receive one of the two treatments for four weeks. Researchers wi…

This study tests whether a supplement called geranylgeraniol (GG), derived from annatto seeds, can reduce muscle pain and weakness caused by statin medications. Researchers will give 95 adults either a low or high dose of GG or a placebo for three months. The goal is to see if GG…

This study tests whether dry needling, a technique where thin needles are inserted into muscle trigger points, can reduce shoulder pain caused by myofascial pain syndrome. Fifty adults with shoulder pain will receive either real dry needling or a sham version, and both patients a…

This study tests whether dry needling—inserting thin needles into muscle trigger points—can reduce back pain and improve daily function. Fifty adults with back pain from myofascial trigger points will receive either real or sham dry needling. Neither they nor the evaluator will k…

This study aims to learn more about rare inherited disorders that affect the brain, spinal cord, muscles, and nerves. Researchers will collect medical history, perform exams, and run genetic tests on up to 3,500 participants. No new treatments are tested; the goal is to better un…

This registry study follows about 100 people with FOP, a rare disease where soft tissue turns to bone. Some participants take palovarotene (an approved treatment), while others do not. Researchers will track side effects, flare-ups, and daily function over time to see how well th…

This study will test how the heart drug empagliflozin is absorbed and processed in 10 children with Duchenne muscular dystrophy, ages 8 to 18. The goal is to find the best dose for protecting the heart in this rare disease. Participants will take the drug by mouth and have blood …

This study screens males with Duchenne muscular dystrophy (ages 0 to under 25) for antibodies against AAV8, a virus used in some gene therapies. The goal is to find out how many have these antibodies and to identify who might be eligible for future gene therapy trials. No treatme…

This study aims to learn more about myositis, a group of diseases that cause muscle inflammation and damage. Researchers will observe up to 1,200 children and adults with myositis, along with healthy volunteers, to track changes in the immune system and other health problems over…

This study aims to understand how heart problems develop in people with Duchenne and Becker muscular dystrophy, as well as in carriers. Researchers will collect cardiac MRI scans and clinical data from 1,000 participants to build a registry. Using advanced image analysis and deep…

This study is testing a new leg exoskeleton designed by the NIH to help children and young adults with muscle weakness from conditions like cerebral palsy or spinal cord injury. Researchers want to see how well the device works in different real-world settings, like walking on a …

This study follows about 50 boys under 18 with X-linked myotubular myopathy (XLMTM), a rare genetic muscle condition, to see how often they develop liver problems like cholestasis. No drugs are given—researchers simply collect health data, including liver scans, over one year. Th…

This study aims to collect medical information and tissue samples from 500 people with suspected or confirmed inflammatory muscle diseases, as well as healthy volunteers. Researchers will use blood, muscle, and skin samples to study what causes these diseases and how they progres…

This study is building a library of information and samples from 60 adults with suspected autoimmune myopathy, a rare muscle disease. Researchers will collect blood, urine, stool, and muscle tissue, along with medical data, to better understand the condition. The goal is to impro…

This study is testing whether a special ultrafast ultrasound can better measure muscle changes in boys and men with Duchenne or Becker muscular dystrophy. Researchers will compare muscle stiffness, fat content, and blood flow in 60 participants (patients and healthy volunteers) o…

This study follows children and adults worldwide who have a rare genetic muscle disease called TNNT1 myopathy. Researchers aim to document how the disease progresses over time, including survival and motor milestones. The goal is to identify reliable measures that can be used in …

This study will test whether acupuncture can improve pain and function in people with chronic low back pain caused by myofascial pain syndrome. Sixty-four adults will receive either real acupuncture or sham (fake) acupuncture for eight weeks. Researchers will use full-body PET/CT…

This study will track jaw muscle activity in 42 adults with chronic jaw pain and teeth grinding (bruxism) who use a custom mouth splint at night. Participants will wear a portable device for 24 hours to measure muscle activity before starting the splint, two weeks later, and afte…

This study is for people with a rare genetic muscle condition called congenital myasthenic syndrome (CMS). It does not test any new drug or treatment. Instead, researchers will collect information from up to 100 participants over 4 visits to better understand how the disease affe…

This study looks at how emotional intelligence—the ability to understand and manage emotions—relates to chronic orofacial pain (pain in the face or mouth lasting over three months). Researchers will compare 88 adults with and without this pain using questionnaires about emotions,…

This study is collecting skin and blood samples from 830 people with chronic inflammatory skin diseases like psoriasis, eczema, and lupus. Researchers want to find biological markers that can predict how well a person will respond to treatment after one year. The goal is to move …

This study is following 700 adults with myotonic dystrophy type 1 (DM1) over two years. Researchers are measuring walking speed, lung function, and muscle tissue changes to find reliable markers of disease progression. The goal is to improve future clinical trials by better under…

This study investigates whether certain environmental exposures—like infections, stress, or sun exposure—are more common in people with anti-synthetase syndrome, a rare autoimmune condition that causes muscle weakness and lung disease. Researchers will compare 580 participants, i…

This study is for children up to 4 years old with congenital myotonic dystrophy type 1 (CDM). Researchers want to find better ways to measure how the disease affects motor skills and language, and to identify biological markers. The goal is to improve future clinical trials and c…

This study is testing whether a watch-like device worn on the ankle can measure how well toddlers with Duchenne muscular dystrophy (DMD) move during their daily activities. Researchers will ask 30 boys with DMD and 30 without (ages 1-3) to wear the device for three 28-day periods…

This study translates and tests several health questionnaires—covering pain, arm and leg function, mood, and daily activities—for use with Albanian-speaking people. Researchers will check if the translated versions are reliable and accurate by giving them to about 300 participant…

This study gathers medical information from people with MNGIE, a rare genetic disease that affects the nerves and digestive system. Researchers want to learn how the disease progresses and find better ways to measure it. Up to 50 patients worldwide can join, and no new treatments…

This study aims to learn why myotonic dystrophy type 1 affects children differently than adults, and why symptoms vary even within the same family. Researchers will observe 100 children (ages 0-17) through video calls and simple at-home activities, and analyze their genes from a …

This study tests a new tool called the PRISM Dashboard, which gives physiotherapists feedback on how their decisions compare to their peers. The goal is to see if this feedback helps reduce differences in care for people with muscle and joint pain. About 60 physiotherapists and t…

This study aims to learn how well heart scans (MRI, ultrasound, PET) can detect and predict outcomes in people with suspected or known inflammatory heart diseases. Researchers will follow 5,000 adults for one year to see how often heart involvement is confirmed and how it affects…

This study follows up to 5,000 people with autoinflammatory diseases (like NOMID, CANDLE, and juvenile dermatomyositis) and their healthy relatives. Researchers will collect medical history, blood samples, and imaging over 2-5 day visits to learn how these diseases work and find …

This study looks at whether a longer fasting period before an upper endoscopy can lower the risk of stomach contents entering the lungs (aspiration) in people taking GLP-1 receptor agonists (like Ozempic or Wegovy). Researchers at Mayo Clinic will check the stomachs of 150 adults…

This early-stage study tests a brain implant called BrainGate in just 3 people with severe paralysis or speech loss from conditions like ALS or spinal cord injury. The goal is to see if the device is safe and can let users control a computer cursor or other tools by thought alone…

This study will collect electronic health records from up to 2500 people with Duchenne or Becker muscular dystrophy, including female carriers, across U.S. clinics. The data will be combined with patient-reported information to give researchers a fuller picture of the diseases. T…

This study uses three wearable devices—a continuous glucose monitor, a heart monitor, and an activity tracker—to see if high blood sugar is linked to heart problems in people with Duchenne muscular dystrophy. Researchers will compare 80 participants with and without DMD. The goal…

This registry collects information from people with inherited retinal diseases, like retinitis pigmentosa and Stargardt disease. Participants share their symptoms, family history, and genetic test results online. The goal is to help researchers understand these rare diseases and …

This study is testing whether a smartphone app can accurately measure how people walk, without needing special markers or equipment. Researchers will compare the app's measurements to a standard motion-capture system in 30 volunteers—some with neuromuscular diseases like SMA or m…

This study looks at blood clotting in people with autoimmune skin diseases like bullous pemphigoid and lupus. Researchers will collect blood samples from 39 participants—some with active disease and some with mild or controlled disease—plus 15 healthy controls. They will measure …

This study follows 450 people with FSHD for at least 3 years to measure how the disease affects walking, arm use, breathing, and other daily activities. The goal is to improve patient care and help researchers design better clinical trials by understanding what changes in movemen…

This study is building a worldwide online registry for people with Duchenne or Becker muscular dystrophy, including female carriers. Participants share their health information to help researchers learn more about the disease and to match patients with clinical trials. The goal i…

This early study tests whether a brain implant can help people with severe paralysis from conditions like ALS, spinal cord injury, or stroke control devices using their thoughts. The implant records brain signals to decode text or synthesized speech. Only 3 adults will participat…

This study follows 105 boys from birth to age 3 who have Duchenne muscular dystrophy (DMD), identified through newborn screening. Researchers will measure motor and cognitive skills over time to understand how the disease develops in early childhood. The goal is to gather informa…

This study tests whether adding servo-assisted bed cycling to standard physical therapy can reduce muscle breakdown in critically ill patients with sepsis. Researchers will measure inflammation and muscle damage markers in 24 participants over 7 days. The goal is to understand if…

This study looks at whether a doctor's computer tool (MedSafer) and patient brochures (EMPOWER) can help reduce the use of potentially harmful medications in adults aged 60 and older with rheumatic diseases who take five or more regular drugs. Researchers will follow 100 particip…

This study explores whether a non-invasive brain-computer interface (BCI) can help people with motor disorders, such as spinal cord injury or stroke, control assistive devices using their thoughts. Researchers will record brain signals with EEG and use machine learning to interpr…

This study uses ultrasound, a safe imaging technique that uses sound waves, to get detailed pictures and measurements of nerves and muscles. Researchers aim to collect normal values from healthy adults and compare them with data from people who have neuromuscular disorders. The g…

This study aims to find better ways to detect lung scarring (interstitial lung disease) early in people with connective tissue diseases like scleroderma or rheumatoid arthritis. Researchers will analyze tiny particles in the blood called extracellular vesicles and use AI to exami…

This study looks back at the last 10 years in Denmark to understand how many people use breathing machines at home through a tube in their windpipe. Researchers will track which diseases lead to this need and how many patients survive one year after starting. The goal is to spot …

This study looks at medical records of 1000 children with conditions like autism, epilepsy, and Down syndrome to find common biological patterns. Researchers want to understand what causes these disorders and how children respond to treatments. The goal is to improve diagnosis an…

This global registry collects information from people with Fibrodysplasia Ossificans Progressiva (FOP), a rare condition where soft tissues turn into bone. Up to 800 patients worldwide can report their symptoms, flare-ups, and mobility changes through a secure online portal. The …

This study watches how children under 5 with LAMA2-related muscular dystrophy move and develop over two years. Researchers will test muscle function, swallowing, breathing, and collect blood samples. The goal is to learn more about the disease so future treatments can be tested e…

This study is testing whether special insoles worn inside shoes can measure how well children with Duchenne muscular dystrophy (DMD) or spinal muscular atrophy (SMA) walk. About 106 participants, including healthy children, will wear the insoles during walking tests and in daily …

This study is testing whether a protein called titin, found in urine, can serve as a reliable marker of muscle injury in people with Duchenne or Becker muscular dystrophy. Researchers will measure titin levels before and after activities like walking down stairs, and track daily …

This study aims to understand how different types of changes in the DMD gene are linked to heart problems in boys and young men with Duchenne or Becker muscular dystrophy. Researchers will use heart tests and blood samples to look for early signs of heart issues. The study involv…

This study looks at whether a movement screening test can accurately detect changes in Achilles tendon pain in runners over 40. Forty senior runners with Achilles tendinopathy will be randomly assigned to two physiotherapy groups, one with extra stretching. The goal is to see if …

This study aims to understand why myotonic dystrophy type 1 affects people so differently. Researchers will remotely assess muscle strength, memory, and activity in 1,000 participants, and analyze their DNA from a blood sample. All activities are done from home using a mailed too…

This study collects information from people with dermatomyositis to better understand how the disease affects their skin and quality of life. Researchers will use a special scoring tool called CDASI to measure skin changes over time. The goal is to improve knowledge about the dis…

This study follows 252 adults with muscle, bone, or joint problems (like arthritis, back pain, or after surgery) to see how well they do their prescribed home exercises. Researchers will track who sticks with the program and why, looking at factors like pain, mood, and support. T…

This research study at Boston Children's Hospital is looking at the genes and proteins involved in congenital myopathies—rare muscle diseases that are present from birth. Researchers will analyze DNA from up to 4,000 participants, including patients and their family members, to f…

This study follows 100 people in Spain with nemaline myopathy, a rare muscle disease, to see how their symptoms change over time. Researchers will use ultrasound, movement tests, and breathing checks to map the disease's natural course. The goal is to build a detailed patient dat…

This study uses ultrasound to track how soft tissues like ligaments, tendons, and muscles stretch and move in different groups: baseball pitchers, people with ankle tendon problems, and patients recovering from ACL knee surgery. Researchers want to see if these measurements can h…

This study aims to create a large collection of blood samples and medical information from 800 people with autoimmune or inflammatory skin diseases. Researchers will use these samples to find new biomarkers and better understand what causes these conditions. The goal is to help d…

This study aims to collect blood samples and medical data from 3000 people with autoimmune diseases like lupus, scleroderma, and myositis. Researchers will use these samples to find new biomarkers and better understand these conditions. The goal is to help develop new treatments …

This study investigates whether serious muscle weakness, known to affect ICU patients, also occurs in people hospitalized outside the ICU. Researchers will follow about 600 adults in Italian hospitals, half with organ failure and half without, to see how often weakness develops a…

This study uses ultrasound imaging and machine learning to better understand low back pain by looking at the thoracolumbar fascia, a layer of tissue in the back. Researchers will compare images from people with acute pain, chronic pain, and no pain. Some participants with chronic…

This study is observing up to 20 children diagnosed with juvenile eosinophilic fasciitis, a rare connective tissue disease. Researchers will track symptoms, lab results, and treatments over time to better understand the condition and improve future care. No new drugs or procedure…

This study tests whether using a virtual reality (VR) headset during shoulder exercises helps patients feel more engaged and motivated. Fifty adults with shoulder problems will do rehab exercises in a VR environment and then answer questionnaires about how easy and immersive the …

This study aims to identify genetic factors that affect how severe muscle and heart problems become in people with LMNA gene mutations. Researchers will collect skin and muscle samples from 40 participants and use advanced DNA and RNA analysis to look for protective or aggravatin…

This study looks at how exercise training changes muscle cells in people with mitochondrial myopathy, a rare disease that affects energy production. Researchers will compare a trained leg to an untrained leg in the same person, and also compare results with healthy volunteers. Th…

This study looks at whether quick, easy walking tests can replace the current complex and time-consuming exam used to track walking problems in people with facioscapulohumeral muscular dystrophy (FSH). Researchers will compare results from simple tests like the 6-minute walk with…

This pilot study is looking at why some patients in the intensive care unit (ICU) develop severe muscle weakness after surgery. Researchers will take blood and small muscle samples from 20 high-risk patients to look for chemical changes that might cause or signal this weakness. T…

This study is testing different ways to measure muscle strength and movement in children with a rare muscle disease called FSHD. The goal is to find the best tests to use in future studies of new treatments. About 80 children aged 5 to 17 with confirmed FSHD will take part. The r…

This study is looking at whether a molecule in the blood called miR-1 can help doctors understand how muscle diseases like Duchenne muscular dystrophy and myotonic dystrophy are progressing. Researchers will compare miR-1 levels in 104 people, including patients with different mu…

This study tests whether taking dietary nitrate (found in beets) can help prevent muscle loss and weakness when a leg is immobilized in a brace for 14 days. Researchers will measure muscle function and energy use in 24 healthy young men. The goal is to find a simple, drug-free wa…

This study creates a registry for people with facioscapulohumeral muscular dystrophy (FSHD) to share their health experiences and data. Up to 5,000 participants in the U.S. will provide information through surveys to help researchers better understand the disease and develop impr…

This study follows about 70 people who have already received KYV-101, a CAR T-cell therapy, for autoimmune diseases like lupus and multiple sclerosis. Researchers will monitor participants for up to 15 years to track any delayed side effects, new cancers, or other health problems…

This study follows 220 boys aged 4 to 9 with Duchenne muscular dystrophy (DMD) for up to 3 years. Researchers collect data on muscle strength, walking ability, heart and lung function, and quality of life to better understand how the disease progresses. The goal is to gather natu…

This study is creating a new classification system for shoulder calcific tendinitis based on MRI scans. Researchers will look at 400 patients to see if the location of calcium deposits relates to which treatment works best. The goal is to help doctors choose between conservative …

This study is building a large registry and biorepository of 5,000 adults with various rheumatic diseases like rheumatoid arthritis, lupus, and scleroderma. Researchers at Yale University will collect medical data and biological samples over time to better understand these condit…

This study aims to find genes that cause eye misalignment (strabismus) and related conditions involving the cranial nerves and brainstem. Researchers will analyze DNA from up to 20,000 participants with these disorders. The goal is to better understand the genetic causes, which c…

This study uses ultrasound to measure muscle, fat, and fluid changes in 121 critically ill pregnant or gynecological patients in the ICU. The goal is to see if these body composition changes are linked to serious outcomes like death, need for a breathing machine, or longer hospit…

This study tracks how quickly the diaphragm weakens in 15 ICU patients on mechanical ventilation. Researchers use advanced muscle tests and brain stimulation to measure changes in diaphragm function and control. The goal is to better understand ventilator-induced diaphragm dysfun…

This study looks at how much protein adults with facioscapulohumeral muscular dystrophy (FSHD) need each day to support muscle health. Researchers will test different amino acid intakes in 10 participants using a non-invasive technique. The goal is to improve dietary recommendati…

This study aims to create a secure national database for muscle and joint (musculoskeletal) conditions by collecting routine data from community clinics and GP practices. About 22,000 adults will have their information anonymously gathered to help develop a dashboard that tracks …

This study looks at how myotonic dystrophy types 1 and 2 affect the brain. About 100 adults aged 30-65 will have MRI scans, thinking and movement tests, and blood draws. Some will also have a spinal tap. The goal is to find brain changes that could be used as markers in future tr…

This study looks at why exercise helps some people with shoulder pain from rotator cuff tendinopathy but not others. Researchers will use brain scans to see how exercise changes the brain and find out who is most likely to get better. The goal is to make exercise treatments more …

This study aims to create a national registry for people with Myotonic Dystrophy Type 1 (DM1) in Spain. Researchers will collect clinical data, genetic information, and patient reports from up to 3,000 participants. The goal is to better understand the disease and identify people…

This study is creating a large registry and tissue bank for people with mitochondrial disorders. Researchers will collect medical information and samples from up to 1,000 participants, including those diagnosed with or suspected to have a mitochondrial disease. The goal is to gat…

This study tests a special MRI method to see how muscles and connective tissues move and respond to pressure. Researchers will compare healthy people and those with myofascial pain, and see if hands-on therapy (Tuina) changes tissue movement. Participants with pain will be random…

This study asks 700 people with rare myositis diseases (and their caregivers) what research questions matter most to them. Through online surveys and forums, the goal is to create a patient-driven list of priorities to guide future studies and funding. No drugs or treatments are …

This observational study looks at saliva thickness and oral health in people with Oculopharyngeal Muscular Dystrophy (OPMD) compared to their household members. Researchers want to see if swallowing problems affect oral health and if saliva contains markers of the disease. No tre…

This study is looking for 75 adults with myotonic dystrophy (a muscle disease) and healthy volunteers to test new muscle imaging techniques. The goal is to find better ways to measure muscle changes over time, which could help future treatment studies. Participants will undergo M…

This study follows 300 people who carry a specific mitochondrial DNA mutation (m.3243A>G) that can cause symptoms like migraines, seizures, and hearing loss. Researchers will use brain scans, muscle tests, and cognitive assessments to track how the disease progresses over time. N…

This study follows 150 people aged 7 and older with RYR1-related muscle disorders for 3 to 5 years. Researchers will collect medical history, perform physical exams, and use questionnaires, activity trackers, and tests of heart, lung, and muscle function. The goal is to better un…

This study watches 2000 people having hip or knee replacement surgery to see how long spinal anaesthesia lasts. The goal is to learn how the amount of anaesthetic affects timing, helping doctors decide if patients can safely go home the same day. No new treatments are tested—just…

This study looks at whether simple measurements—like thigh circumference and muscle stiffness—can reliably track muscle health in people with COPD. Researchers will measure 30 patients before and after a pulmonary rehabilitation program. The goal is to find an easy way to monitor…

This study aims to find better ways to measure fatigue and walking problems in people with neuromuscular diseases like muscular dystrophy and spinal muscular atrophy. Researchers will use a wearable sensor to track physical activity for one week in daily life and during a walking…

This study collects DNA from up to 1,000 adults with progressive supranuclear palsy (PSP), corticobasal syndrome (CBS), multiple system atrophy (MSA), or related conditions, plus their family members. Researchers will sequence participants' whole genomes to find genetic variants …

This study aims to describe a rare condition called panniculitis (inflammation of the fat under the skin) in people with dermatomyositis. Researchers will examine 20 participants aged 1 year and older to identify unique clinical and microscopic features. The goal is to better dis…

This study is a registry that collects health information from people in Switzerland who have neuromuscular disorders like SMA, DMD, BMD, and others. It aims to track symptoms, treatments, and outcomes over time to help researchers and doctors improve care. No new treatments are …

This registry enrolls pregnant women or those planning pregnancy who have autoimmune diseases like lupus or rheumatoid arthritis. Researchers at Duke University will track health data to find ways to improve outcomes for mothers and babies. The goal is to better understand risk f…

This study aims to find better ways to measure myofascial pain, a common type of muscle pain. Researchers will use MRI and other imaging tools to look at muscle tissue in 100 adults with neck or shoulder pain. The goal is to identify imaging markers that can track how well treatm…

This study tracks 200 people with ultra-rare glycogen storage diseases (GSDs) like types 0a, 0b, VII, X, XII, XIII, XV, PRKAG2 syndrome, and Danon disease. Researchers will collect medical records and other data to see how these conditions progress over time. No treatments or dru…

This study follows 100 adults who needed ECMO support for severe heart or lung failure. Researchers will track muscle size, strength, and quality of life over time to understand why some patients develop lasting weakness. No new treatments are being tested; the goal is to gather …

This study looks at how changing the heel cushion on an ankle-foot brace (AFO) affects walking in people who use one daily. Forty adults with leg injuries or nerve problems will try four different heel wedges—tall or short, soft or firm—while walking at controlled speeds. Researc…

This study tests whether a special ultrasound test (called the NUN biomarker) can track changes in chronic low back pain after dry needling and predict who will feel better. Researchers will compare real dry needling to a sham (fake) procedure in 80 adults with muscle-related bac…

This study looks at how 3D computer simulation and printing can help surgeons better plan and perform bone correction surgeries. Researchers will compare the planned corrections to the actual results in 100 children and young adults with rare bone deformities. The goal is to make…

This study is not a treatment trial. It aims to collect blood samples from 400 children and their relatives who have rare autoimmune or autoinflammatory diseases (like lupus or juvenile arthritis). Researchers will analyze the samples to find genetic mutations that cause these co…

This study uses special wearable devices to monitor how babies and children with spinal muscular atrophy (SMA) or Duchenne muscular dystrophy (DMD) move at home. About 100 children will wear a sensor-filled jumpsuit or an ankle monitor to track their motor skills over up to 30 mo…

This study tracks 10 people in Guadeloupe with dermatomyositis, a rare autoimmune disease that affects skin and muscles. Researchers want to see how severe the disease is at diagnosis, including muscle weakness, organ problems, and cancer links. No new treatment is being tested —…

This study aims to understand how muscle health recovers after a period of disuse, like being in a cast. Researchers will immobilize one leg of healthy volunteers for two weeks, then measure muscle size and strength before and after. The study includes both younger (18-35) and ol…

This study aims to find a way to predict which cancer patients will experience side effects from immunotherapy. These side effects happen when the immune system attacks healthy tissues, similar to autoimmune diseases. Researchers will collect blood samples from 300 cancer patient…

This study tests a brain implant that reads signals from the brain to help people who have lost the ability to speak fluently communicate through a computer just by trying to talk. It involves up to 2 participants with conditions like spinal cord injury or ALS. The main goal is t…

This study aims to learn how the binding of RNA with DNA (called R-loops) is linked to amyotrophic lateral sclerosis type 4 (ALS4) and other inherited neurological disorders. Researchers will observe up to 330 people aged 5 and older, including those with ALS4, related conditions…

This study aims to find less invasive ways to measure Duchenne muscular dystrophy activity. Researchers will collect urine and blood samples from 100 participants, including boys with Duchenne or Becker muscular dystrophy and healthy adults. They will look for RNA biomarkers that…

This study looks at the spinal fluid and brain activity of people with myotonic dystrophy type 1, a condition that affects muscles and thinking. Researchers want to find early signs of disease in the fluid that surrounds the brain. About 88 adults will take part, including some w…

This study aims to find less invasive ways to measure muscle disease activity in people with muscular dystrophies. Instead of painful muscle biopsies, researchers will use blood and urine samples along with painless ultrasound and electrical tests on the arms and legs. The goal i…

This study aims to find less invasive ways to measure disease activity in myotonic dystrophy by looking for RNA markers in blood and urine instead of taking muscle biopsies. Researchers will compare samples from 215 people with and without the condition to see if these markers ca…

This study follows 4,000 children who receive orthopedic implants (like rods or screws) during standard care for bone fractures, deformities, or hip problems. Researchers will track how long the implants last, any complications, and how well they work over time. The goal is to ga…

This study aims to find genetic differences that make some cancer patients more likely to develop a rare but life-threatening heart inflammation (myocarditis) from immune checkpoint inhibitors. Researchers will compare the DNA of 100 patients who had this side effect with 400 who…

This study follows 144 adults with muscle or bone problems who are already scheduled to receive treatment with Chattanooga Intelect devices (like TENS or ultrasound). Researchers will measure pain, muscle strength, and movement range to confirm the devices are safe and work as ex…

This study looks at how muscle wasting in the mouth and throat affects swallowing in adults who are critically ill and in the intensive care unit. Researchers will measure muscle size and strength, and test swallowing safety at several points during the hospital stay. The goal is…

This 24-month observational study will follow up to 1000 people with certain types of muscular dystrophy (LGMD, DM2, and late-onset Pompe disease) aged 6-50. Researchers want to see if specific physical tests, like the North Star Assessment and a 100-meter walk, are good ways to …

This study looks at whether a special bed that gently moves your legs (passive exercise) can improve how the body uses oxygen in people with mitochondrial disease. Researchers will compare patients to healthy volunteers and also test the bed in children in the ICU. The goal is to…

This study will test if wearable devices like continuous glucose monitors and heart rate monitors can help detect early signs of heart problems in people with Duchenne muscular dystrophy (DMD). Ten participants will wear these devices at home over two years and have periodic hear…

This study tests whether a type of exercise using a flywheel machine can help prevent muscle loss and weakness during space travel. Sixteen healthy adults who exercise regularly will take part. They will use crutches and a special shoe to mimic the reduced weight-bearing of space…

This study uses MRI scans to track muscle damage and fat buildup in boys and men with Duchenne or Becker muscular dystrophy over 5-10 years. Researchers will compare these images with how well participants can walk and perform daily activities. The goal is to find better ways to …

This registry aims to connect people diagnosed with myotonic dystrophy (DM) or facioscapulohumeral muscular dystrophy (FSHD) with researchers. By joining, participants help scientists better understand these inherited muscle-weakening diseases and develop future treatments. The r…

This study looks at what happens to muscle size, strength, and insulin sensitivity after 5 days of strict bedrest in healthy young adults. Twelve volunteers will undergo tests before and after the bedrest period, including MRI scans, strength tests, and muscle biopsies. The goal …

This study follows 150 people with primary mitochondrial myopathy, a rare genetic muscle disease, to understand how the condition changes over time. Researchers will measure muscle function, biomarkers, and imaging to find signs of disease progression. The goal is to identify use…

This study tests whether taking 20 or 40 grams of whey protein can help prevent muscle loss when a leg is immobilized in a knee brace. Researchers will measure how well muscles build protein in 28 healthy adults. The goal is to find the best protein dose to keep muscles strong du…

This observational study aims to better understand primary mitochondrial myopathy, a rare muscle disease. Researchers will follow 1300 patients and healthy controls, measuring muscle strength, balance, and daily function over time. The goal is to develop and validate tools to tra…

This study aims to find the genes that cause inherited heart and muscle diseases like cardiomyopathy, arrhythmias, and muscular dystrophy. Researchers will collect blood samples from up to 2,000 patients and their families to look for genetic mutations. The goal is to better unde…

This study looks at how brain circuits are connected in people with chronic temporomandibular joint (TMD) pain compared to pain-free individuals. Researchers will use MRI scans to map brain activity and structure in 95 female participants. The goal is to find patterns that could …

This study follows 34 adults with FSHD over one year to see how their breathing muscles change. Researchers will use ultrasound to measure diaphragm thickness and movement, along with standard breathing tests. The goal is to find out if ultrasound can detect respiratory problems …

This study aims to learn more about rare disorders that affect how the body processes chemicals called pyrimidines and purines. These disorders can cause problems in the brain, blood, kidneys, and immune system, ranging from mild to life-threatening. Researchers will compare test…