DUCHENNE MUSCULAR DYSTROPHY

This early-stage trial tested a gene therapy called scAAV9.U7.ACCA in 3 boys with Duchenne muscular dystrophy caused by a duplication of exon 2. The therapy was given as a single injection into a vein. The main goals were to check for serious side effects and to see if the treatm…

This study tested two experimental drugs, casimersen and golodirsen, in 228 people with Duchenne muscular dystrophy (DMD) who have specific genetic mutations. The goal was to see if these drugs could help maintain muscle function, such as walking speed and distance, compared to a…

This study tested a drug called vamorolone in 54 boys with Duchenne muscular dystrophy (DMD), a muscle-weakening disease. The goal was to see if the drug is safe and can help control the disease with fewer side effects than standard steroids. Boys aged 2 to under 4 and 7 to under…

This study looked at the safety of a drug called ataluren in 270 people with Duchenne muscular dystrophy caused by a specific genetic mistake (nonsense mutation). Participants had already taken ataluren in earlier studies or were siblings of those who did. The goal was to see how…

This study tested whether nebivolol, a beta-blocker, could prevent heart muscle weakness in boys aged 10–15 with Duchenne muscular dystrophy. Participants had normal heart function at the start and were randomly assigned to receive nebivolol or a placebo. The trial found that neb…

This study looked at whether virtual reality (VR) and biofeedback training can improve movement and motivation in 24 children with neuromuscular diseases like Duchenne muscular dystrophy and spinal muscular atrophy. Children were split into three groups: VR training, biofeedback …

This study tested whether special breathing exercises, done at home with a therapist over video calls, could improve lung function and quality of life in 40 children with Duchenne muscular dystrophy (ages 5-17). The exercises focused on strengthening the muscles used to breathe. …

This study looked at whether an interactive virtual reality (VR) system could help children with Duchenne muscular dystrophy (DMD) stick to their physiotherapy exercises at home. Fourteen children aged 5-10 who could walk took part. The study measured how often and how long they …

This study enrolled 150 people with Duchenne Muscular Dystrophy (DMD) to create a video database of their movement abilities at home. Caregivers recorded 18 specific tasks using a secure app, and physical therapists scored the videos to validate a new assessment tool called the D…