DUCHENNE MUSCULAR DYSTROPHY

This study tested the safety of a drug called ataluren (also known as Translarna) in 270 people with Duchenne muscular dystrophy caused by a specific type of genetic mistake (nonsense mutation). Participants had already taken ataluren in earlier studies. The goal was to monitor s…

This study tested two drugs, casimersen and golodirsen, in 228 boys with Duchenne muscular dystrophy who have specific genetic mutations. The goal was to see if these drugs could slow muscle decline, measured by how fast they could climb four steps. Participants received either t…

This study tested a drug called vamorolone in 54 boys with Duchenne muscular dystrophy, aged 2 to under 4 years and 7 to under 18 years. The goal was to check safety and how the body processes the drug over 12 weeks. Researchers hope vamorolone may help control the disease with f…

This early-stage trial tested a single-dose gene therapy called scAAV9.U7.ACCA in 3 boys with Duchenne muscular dystrophy caused by a duplication of exon 2. The therapy was given through a vein and aimed to help muscle cells produce dystrophin, a protein missing in this disease. …

This study tested whether nebivolol, a beta-blocker heart drug, could prevent heart failure in 51 boys aged 10-15 with Duchenne muscular dystrophy. Participants had normal heart function at the start and were randomly given either nebivolol or a placebo. The trial found that nebi…

This study tested whether virtual reality (using Xbox Kinect) or biofeedback training could improve motor function and balance in 24 children with neuromuscular diseases like Duchenne muscular dystrophy and spinal muscular atrophy. Participants were split into three groups: VR tr…

This study tested whether an interactive virtual reality (VR) system could help children aged 5–10 with Duchenne muscular dystrophy (DMD) stick to their physiotherapy exercises at home. Fourteen children used the VR system for 8 weeks, and researchers measured how often and how l…

This study tested whether breathing muscle exercises done at home via video calls could improve lung function and quality of life in 40 children with Duchenne muscular dystrophy. The children, aged 5-17, used a device to strengthen their breathing muscles. Researchers measured ch…

This study looked at the extra costs that families of people with Duchenne muscular dystrophy (DMD) in France have to pay out of their own pockets. It included 28 patients of any age who had been diagnosed for at least six months. The researchers measured things like uninsured me…

This study tested whether doctors can accurately assess physical function and quality of life in children with Duchenne muscular dystrophy (DMD) through video calls. Twenty ambulatory patients aged 5-18 completed timed tests and questionnaires remotely. The goal was to see if tel…

This completed study enrolled 150 people with Duchenne muscular dystrophy to create a video database of their movement abilities. Caregivers recorded children doing specific tasks at home using a secure app. The goal was to help validate a new tool called the Duchenne Video Asses…