Skeletal muscle disorder

This early study tested a new cell therapy called LUCAR-G79 in just 3 adults with severe autoimmune diseases that did not respond to standard treatments. The therapy uses specially engineered immune cells to target and potentially calm the overactive immune system. The main goals…

This study tests whether an additional dose of a COVID-19 vaccine can improve immune protection in adults and children with autoimmune diseases like rheumatoid arthritis, lupus, multiple sclerosis, and others who are taking immunosuppressive medications. Participants who had a we…

This study looked at a shoulder implant used to treat pain and joint problems from conditions like arthritis, fractures, and rotator cuff tears. It involved 67 adults who received the implant and tracked how long it lasted and any side effects over 10 years. The goal was to confi…

This study tested whether taking the steroid oxandrolone for 12 weeks after rotator cuff repair could improve tendon healing and muscle mass. Only 4 people participated before the trial was stopped early. Because it was so small, we cannot draw any reliable conclusions about whet…

This phase 2 trial tested two doses of an oral drug called fidrisertib (IPN60130) in children and adults with fibrodysplasia ossificans progressiva (FOP), a rare condition where soft tissues turn into bone. The goal was to see if the drug could reduce new abnormal bone growth com…

This study tested the safety of golodirsen (Vyondys 53) in boys and men with Duchenne muscular dystrophy who can no longer walk. Only 2 people took part before the trial was stopped early. Participants received weekly IV infusions for up to 96 weeks, with extra follow-up. The goa…

This study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of …

This study tested a drug called OMS721 in 31 people with certain kidney diseases, including IgA nephropathy and lupus nephritis. The main goal was to check if the drug is safe and if it reduces protein in the urine, a sign of kidney damage. The study was stopped early, so results…

This early-phase trial tested a single infusion of IMPT-514, a personalized CAR T cell therapy, in 3 adults with severe lupus, vasculitis, or inflammatory myopathy that did not respond to standard treatments. The goal was to check safety and see if the therapy could reset the imm…

This study tested a gene therapy called SRP-9003 for people with limb-girdle muscular dystrophy type 2E (LGMD2E), a rare genetic disease that causes muscle weakness. The treatment aimed to deliver a working gene to muscle cells to help them produce a missing protein. Only 6 peopl…

This study tested a medicine called Hizentra (IgPro20) in 134 adults with dermatomyositis, a rare disease causing muscle weakness and skin rashes. The goal was to see if weekly shots under the skin could improve symptoms better than a placebo. The study was stopped early, but res…

This study tested an experimental drug called losmapimod in 14 adults with FSHD1, a rare genetic condition that causes progressive muscle weakness. The main goal was to check safety and tolerability, and to see if the drug affects certain biological markers. The trial was termina…

This phase 2 study tested a drug called PGN-EDO51 in 7 people with Duchenne muscular dystrophy whose genetic mutation can be corrected by skipping exon 51. The drug was given by IV infusion to see if it is safe and tolerable. The trial was terminated, so results are limited.

This study tested a gene therapy (delandistrogene moxeparvovec) combined with a drug called imlifidase to see if it could safely deliver the therapy to boys with Duchenne muscular dystrophy who had antibodies that might block the treatment. Only 5 participants were planned, but t…

This study tested a drug called losmapimod for people with a rare muscle-weakening disease called FSHD. The goal was to see if the drug could slow muscle loss and improve arm function over 48 weeks. About 260 adults with FSHD were randomly assigned to receive either losmapimod or…

This study tested whether cutting a specific tendon (the conjoint tendon) during reverse shoulder replacement surgery helps patients move their hand behind their back better. The trial planned to enroll 26 adults having their first reverse shoulder replacement, but it was termina…

This Phase 2 trial tested a single dose of gene therapy (fordadistrogene movaparvovec) in 10 boys with early-stage Duchenne muscular dystrophy. The goal was to check safety and whether the therapy could help muscles produce a mini-dystrophin protein. The study was terminated earl…

This study follows 7 people with Duchenne muscular dystrophy who previously received an experimental gene therapy called fordadistrogene movaparvovec. Researchers will monitor them for 10 years to check for side effects and see if the treatment continues to help with movement. Th…

This phase 2 trial tested the drug losmapimod in 76 adults with FSHD, a genetic condition that causes progressive muscle weakness. Participants took either losmapimod or a placebo for 48 weeks to see if the drug was safe and could help control the disease. The study was terminate…

This early-stage trial tested a single infusion of gene therapy (PF-06939926) in 23 people with Duchenne muscular dystrophy, both those who could still walk and those who could not. The main goal was to check safety and tolerability, while also measuring dystrophin protein levels…

This early study tested whether a blood-cleaning procedure called plasmapheresis could allow boys with Duchenne muscular dystrophy who have antibodies against the gene therapy carrier to still receive the treatment. Only 3 boys were enrolled before the study was stopped early. Th…

This was a very early (Phase 1) study testing a gene therapy called SRP-6004 for people with limb girdle muscular dystrophy type 2B/R2, a rare muscle-weakening disease. The goal was to see if a single IV infusion of the therapy is safe and can help the body produce a missing prot…

This early-stage trial tested a gene therapy called SRP-9004 in just 4 people with limb girdle muscular dystrophy type 2D/R3, a rare muscle-weakening disease. The main goal was to check safety, not effectiveness. The study was terminated early, so results are limited.

This study tested the drug nintedanib (Ofev) in 11 people with myositis-related interstitial lung disease, a condition where lung scarring occurs alongside muscle inflammation. The goal was to see if the drug is safe and tolerable when added to standard immune-suppressing treatme…

This early-phase study tested a new radioactive imaging drug called 11C-MC1 to see if it can help detect inflammation in people with rheumatoid arthritis or idiopathic inflammatory myopathy, as well as in healthy volunteers. Participants received PET/CT scans after an injection o…

This study tested a medicated patch containing ketoprofen (a pain reliever) for people with tendonitis or bursitis pain in the shoulder, elbow, or knee. About 381 adults with these conditions were randomly assigned to receive either the active patch or a placebo patch. The goal w…

This study tested whether the drug secukinumab could reduce pain and improve function in adults with moderate to severe rotator cuff tendinopathy, a common shoulder condition. Participants received either the drug or a placebo for 24 weeks. The trial was terminated early, and res…

This study looked at whether adding blood flow restriction training to standard physical therapy helps people regain knee strength and muscle size after meniscus repair surgery. About 15 participants were split into two groups: one did standard rehab, the other added the special …

This study looked at whether cutting a small nerve in the wrist during surgery to remove a ganglion cyst helps reduce pain and improve hand function more than just removing the cyst alone. Only 3 adults with painful wrist cysts took part before the study was stopped early. The re…

This study tested the drug secukinumab against a placebo in 60 adults with moderate to severe rotator cuff tendinopathy, a painful shoulder condition. The goal was to see if secukinumab could improve symptoms and shoulder function over 24 weeks. However, the trial was terminated …

This study tested whether using a blood flow restriction (BFR) cuff during physical therapy could help people with shoulder injuries regain strength and return to activity faster. Only 3 people took part before the study was stopped early. The approach uses a special tourniquet t…

This study tested whether two gut hormones, GIP and GLP-2, could reduce bone breakdown in children with spinal muscular atrophy, cerebral palsy, or Duchenne muscular dystrophy who use wheelchairs. Participants received a liquid meal and then either a hormone injection or a placeb…

This study looked at whether muscle stem cells (mesoangioblasts) from people with muscle loss due to cancer or aging are healthy enough to be used for future cell therapy. Researchers took small muscle samples and blood from 7 participants and measured how well the cells grew and…

This study looked at two different ways surgeons place the top part of a reverse shoulder replacement: sitting on top of the bone (onlay) or set into the bone (inlay). The goal was to see if one method leads to better arm movement after surgery. The study enrolled 19 people with …

This study compared three different rehabilitation programs after arthroscopic rotator cuff repair. Participants were divided into groups with varying periods of immobilization and passive motion before starting active rehab. The goal was to see which program leads to the best sh…

This study collected bone, cartilage, skin, and fat tissue from 50 patients undergoing surgery at Children's National Medical Center. The goal was to grow cells from these tissues in the lab and analyze them to better understand bone and connective tissue diseases. The study was …

This study looked at one person with facioscapulohumeral muscular dystrophy (FSHD) to better understand the disease. Researchers examined muscle tissue and checked for specific biomarkers. The goal was to learn more about how FSHD affects the body, not to test a treatment.