Duchenne muscular dystrophy

This early-stage trial is testing a drug called BMN 351 in 18 boys aged 4 to 10 with Duchenne muscular dystrophy who have a specific genetic change. The drug is designed to help the body produce a shorter but still useful version of the muscle protein dystrophin. The main goal is…

This program allows eligible patients to receive RGX-202, a gene therapy, on a single-patient basis outside of a clinical trial. It is designed for those with serious conditions who have no other treatment options. Currently, the program is temporarily not available.

This early-stage trial is testing a drug called NS-050/NCNP-03 in 20 boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The drug is designed to skip a faulty section of the dystrophin gene, allowing the body to produce a shorter but still functional…

This Phase 3 trial tests a cell therapy called deramiocel (CAP-1002) in 106 boys and young men with Duchenne muscular dystrophy. Participants receive either the cell therapy or a placebo every 3 months for a year, then all can receive the therapy for another year. The goal is to …

This study tests a single dose of SGT-001 gene therapy in 12 boys (children and teens) with Duchenne muscular dystrophy. The main goal is to check safety and how well the body tolerates the treatment. Participants will be followed for about 5 years to monitor side effects and any…

This Phase 3 trial tests a gene therapy called SRP-9001 for Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. It includes 148 males who can and cannot walk. Participants receive a one-time IV infusion of the therapy or a placebo, and are followed for about 128 w…

This phase 2 trial tests a daily oral drug called sevasemten (EDG-5506) in 244 adults and adolescents with Becker muscular dystrophy, a condition that causes progressive muscle weakness. The study compares the drug to a placebo to see if it can reduce muscle damage, improve walki…

This study tests satralizumab, a drug that calms inflammation, in 30 children aged 8 to 17 with Duchenne muscular dystrophy. The goal is to see if it improves bone density and muscle function. Participants receive injections for several months while continuing standard steroid th…

This study is for people with Duchenne muscular dystrophy (DMD) who have already taken part in a previous trial of BMN 351. It tests the long-term safety of weekly intravenous infusions of BMN 351 and whether it helps maintain physical function. Only 18 participants will be enrol…

This study tests a gene therapy called delandistrogene moxeparvovec in 13 children under age 4 with Duchenne muscular dystrophy. The goal is to see if it is safe and can help produce a key muscle protein. The children will be followed for about 5 years.

This study looks at the long-term safety and effects of a drug called EDG-5506 (sevasemten) in people with Becker muscular dystrophy, a muscle-weakening disease. About 260 adults and teens who were in earlier EDG-5506 studies will take the drug and be monitored for side effects a…

This study follows 500 people with Duchenne muscular dystrophy to see how well a gene therapy (ELEVIDYS) works compared to standard steroid treatment over time. Researchers will measure movement abilities, breathing, and safety, including liver problems. Participants must already…

This pilot study tests a device called Travagus One, which stimulates a nerve in the ear to reduce inflammation in boys with Duchenne muscular dystrophy (DMD). Twenty boys aged 5-17 will use the headset at home for 5 minutes twice a day for one week. Researchers will measure infl…

This study follows about 80 boys with Duchenne muscular dystrophy who have already taken vamorolone in earlier studies. Researchers want to see how safe the drug is over a longer time, especially its effect on bone health, like spine fractures. The boys continue taking vamorolone…

This study looks at the long-term safety and effects of a drug called SAT-3247 in 10 people with Duchenne muscular dystrophy who were in a previous trial. Participants take the drug by mouth five days a week for about 11 months. Researchers will check for side effects and measure…

This early-phase trial tests the safety and tolerability of a new drug called GRT6019 in 24 healthy men. Participants receive multiple doses over 4 weeks and are monitored for side effects and how the drug moves through the body. The goal is to see if GRT6019 is safe enough to st…

This study follows 66 boys with Duchenne muscular dystrophy who previously received RGX-202 gene therapy. Researchers will monitor side effects and measure muscle function over time, such as how fast they can stand, walk, or climb. The goal is to see if the treatment remains safe…

This early-stage trial tests a single dose of a gene therapy called JWK007 in 3 boys aged 5-10 with Duchenne muscular dystrophy. The main goal is to check safety and tolerability, while also looking at whether it helps muscle function. It is a very small, first-in-human study, so…

This study looks at the long-term safety of an experimental drug called WVE-N531 in people with Duchenne muscular dystrophy (DMD) who have already taken it in a previous study. About 175 participants will receive the drug and be monitored for side effects, heart and lung function…

This study tests a gene therapy called PF-06939926 in 114 boys with Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. Two-thirds receive the gene therapy, while one-third get a placebo, but can switch to the real treatment after one year. The main goal is to see…

This Phase 3 trial tests whether higher doses of eteplirsen (Exondys 51) can safely improve muscle function in boys with Duchenne muscular dystrophy whose genetic mutation allows exon 51 skipping. About 160 ambulatory boys will receive one of three doses (30, 100, or 200 mg/kg) t…

This early-phase trial tests a new drug called RAG-18 in 12 boys aged 4-15 with Duchenne muscular dystrophy. The drug is a small activating RNA given through an IV, designed to help muscles work better. The main goal is to check safety and how the body handles the drug, with earl…

This study tests an experimental drug called sevasemten in 76 children aged 4-9 with Duchenne muscular dystrophy. The goal is to check the drug's safety, how the body processes it, and its effect on muscle health markers. Participants are randomly assigned to receive the drug or …

This study tests an experimental drug called EDG-5506 (sevasemten) in 43 children and teens aged 6 to 17 with Duchenne muscular dystrophy who have already received gene therapy. The goal is to see if the drug is safe and how it affects muscle damage markers. Participants are rand…

This study tests the long-term safety and effectiveness of a cell therapy called deramiocel (CAP-1002) in people with Duchenne muscular dystrophy who completed the earlier HOPE-2 trial. Participants receive an infusion of deramiocel every 3 months for about 5 years, with the opti…

This study tests the long-term safety and effectiveness of an experimental drug called DS-5141b (Renadirsen) in 8 people with Duchenne muscular dystrophy who have already completed a previous study. Participants receive a weekly injection under the skin. Researchers will monitor …

This Phase 3 trial tests whether TAS-205, an oral drug, can improve movement and safety in people with Duchenne muscular dystrophy. It includes 104 participants, both those who can walk and those who cannot. The study compares TAS-205 to a placebo over 52 weeks.

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study tests whether a 6-week, caregiver-assisted breathing program done at home with remote coaching is practical and helpful for children with Duchenne muscular dystrophy. Twelve children will do breathing exercises, airway clearance, and relaxation techniques at least 5 ti…

This study tests whether a dual-task training program—combining physical exercises like walking with mental tasks like counting—can improve both motor and cognitive function in 16 boys aged 6–12 with Duchenne muscular dystrophy. Participants will be randomly assigned to either st…

This 24-month observational study will follow 80 people with Becker muscular dystrophy (BMD) to better understand how the disease progresses. Researchers will measure muscle strength, walking speed, breathing, and heart function using standard tests. The goal is to identify which…

This study tests new radio frequency coils for MRI scanners to see if they can produce better images in children, including those with Duchenne muscular dystrophy. Researchers will check for side effects like heating and discomfort, and measure image quality. The goal is to impro…

This study looked at whether having more frequent contact with a clinician (at least once a week) improves satisfaction for people with long-lasting musculoskeletal conditions. Participants were split into two groups: one with standard contact and one with extra check-ins via tex…

This study tests whether a Norwegian clinic (NSAC) helps people with common mental health issues or muscle pain return to work sooner. 2500 adults are split into three groups: one gets treatment quickly, one waits 10-14 weeks, and one gets a basic check-up. Researchers will track…

This study is testing a virtual reality game designed specifically for people with Duchenne muscular dystrophy (DMD). Six participants aged 7 and older will play the game once using a VR headset and hand tracking. Researchers will measure how easy and enjoyable the game is, and w…

This study looks back at the medical records of about 4 million people who had physical or occupational therapy for muscle and joint problems. Researchers want to see if different ways of giving therapy lead to different results. No new treatments are tested—the goal is to learn …

This study aimed to see if a mobile monitoring tool could accurately identify surgical site infections (SSIs) in orthopedic surgery patients, compared to the usual manual review. The study was withdrawn before enrolling any participants, so no results are available. It was design…

This study looks at how the muscles in the arms and hands change in people with Duchenne muscular dystrophy (DMD). Researchers will measure muscle tone, stiffness, and elasticity using a device called MyotonPRO. They will also test hand function and muscle strength. The goal is t…

This study looks at how thinking skills (called executive functions) relate to everyday activities like self-care and play in children with Duchenne muscular dystrophy (DMD). Researchers will compare 38 children with DMD to healthy children using surveys and functional tests. The…

This study follows about 400 people with Duchenne muscular dystrophy who have already received the gene therapy SRP-9001 in an earlier study. The goal is to monitor their safety and muscle function over time. No new treatment is given in this study.

This study collects blood or skin samples from 100 adults with inherited heart rhythm disorders (like Long QT Syndrome or Brugada Syndrome) and healthy volunteers. Researchers will turn these samples into stem cells and then into heart cells to study how these diseases work and t…

This study was designed to track heart muscle changes in people with Duchenne muscular dystrophy using two cardiac MRIs taken two years apart, along with blood tests for heart failure markers. It planned to enroll participants aged 6 and older with a confirmed genetic diagnosis. …

This observational study will follow 300 people with Duchenne muscular dystrophy who are already taking exon-skipping therapies (eteplirsen, golodirsen, or casimersen) as part of their routine care. Researchers will collect data on movement, lung function, and heart function over…