DUCHENNE MUSCULAR DYSTROPHY

This study follows 500 people with Duchenne muscular dystrophy to see how well a gene therapy (ELEVIDYS) works compared to usual steroid treatment. Researchers will track movement, breathing, and safety over time. Participants must already be on or starting steroids, and those ge…

This study tests the long-term safety and effectiveness of an experimental drug called DS-5141b in people with Duchenne muscular dystrophy (DMD). It includes 8 participants who have already completed an earlier study of the same drug. Researchers will monitor side effects and mea…

This study tests a gene therapy (delandistrogene moxeparvovec) in children under 4 with Duchenne muscular dystrophy, a severe muscle-weakening disease. The goal is to see if the treatment is safe and helps produce a key muscle protein. About 13 children will take part and be foll…

This study tests a gene therapy called PF-06939926 in 114 boys with Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. Two-thirds of participants receive the gene therapy, while one-third get a placebo (with the option to switch to gene therapy after one year). T…

This early-stage trial tests a single dose of a gene therapy called SGT-001 in 12 boys with Duchenne muscular dystrophy (DMD). The main goal is to see if the treatment is safe and tolerable. Participants will be followed for about 5 years to monitor side effects and any changes i…

This study is for people with Duchenne muscular dystrophy who already completed the HOPE-2 trial. It tests the long-term safety and effectiveness of a drug called deramiocel, given as an IV infusion every three months for up to five years. The goal is to see if the drug can help …

This study looks at the long-term safety and effectiveness of vamorolone in boys with Duchenne muscular dystrophy (DMD) who have already taken the drug in earlier studies. About 80 boys will be followed to see how the medicine affects their bones, including the number of spine fr…

This study tests a drug called TAS-205 in 104 people with Duchenne muscular dystrophy, a genetic disease that weakens muscles over time. The goal is to see if the drug can help them move more easily, like standing up from the floor or walking. Participants are split into two grou…

This study tests an experimental drug called EDG-5506 (sevasemten) in 43 children 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 health markers. Participants will first receive…

This study tests an experimental drug called NS-050/NCNP-03 in 20 boys aged 4 to 16 with Duchenne muscular dystrophy (DMD), a genetic condition that causes progressive muscle weakness. The drug aims to help muscles produce a needed protein by skipping a faulty section of the gene…

This study looks at the long-term safety of an experimental drug called WVE-N531 in people with Duchenne muscular dystrophy 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, and …

This study tests a drug called sevasemten in children aged 4-9 with Duchenne muscular dystrophy. The goal is to see if it is safe and how it affects muscle health. Some children get the drug, others get a placebo, and later all can receive the drug. The study is active but not cu…

This early study tests a single infusion of a drug called JWK007 in 3 boys aged 5 to 10 with Duchenne muscular dystrophy. The main goal is to see if the drug is safe and tolerable. Researchers will also check if it helps with muscle function, but this is not a cure and ongoing ma…

This study tests a drug called satralizumab in 30 children (ages 8 to 17) with Duchenne muscular dystrophy who are already taking steroids. The goal is to see if the drug can improve bone density and is safe. The study is active but not recruiting new participants.

This study follows 66 boys with Duchenne muscular dystrophy who previously received RGX-202 gene therapy in an earlier trial. Researchers will monitor safety and measure how well the therapy helps with standing, walking, and climbing over time. The goal is to see if the treatment…

This study follows 300 people with Duchenne muscular dystrophy who are already taking eteplirsen, golodirsen, or casimersen in everyday medical care. Researchers will collect information on movement, breathing, and heart function over time to see how these treatments work in the …

This study looks at how Duchenne muscular dystrophy (DMD) affects children's thinking skills (like memory and planning) and their ability to do everyday tasks such as self-care, schoolwork, and play. Researchers will compare 38 children with DMD to healthy children of the same ag…

This study tracks 400 people with Duchenne muscular dystrophy who previously received the gene therapy SRP-9001. Researchers will monitor their health and muscle function over time to understand long-term safety and effectiveness. No new treatment is given in this study.

This study was designed to observe how heart muscle changes over time in people with Duchenne muscular dystrophy using cardiac MRI and blood tests. It planned to include children and adults aged 6 and older with a confirmed genetic diagnosis. The goal was to better understand hea…