DUCHENNE MUSCULAR DYSTROPHY

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 it 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 18 to…

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, like climbing stairs and walking, compared to a p…

This study tested the safety of a drug called ataluren in 270 people with Duchenne muscular dystrophy caused by a specific genetic mistake (nonsense mutation). Participants took the drug three times daily at different doses. The main goal was to check for side effects and lab cha…

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 whether nebivolol, a beta-blocker, could prevent heart problems in 51 boys aged 10–15 with Duchenne muscular dystrophy. Participants had healthy hearts at the start and were randomly given either the drug or a placebo. The goal was to see if the drug could stop …

This study looked at whether an interactive virtual reality (VR) system could help children aged 5-10 with Duchenne muscular dystrophy (DMD) do their physiotherapy exercises more regularly. Fourteen children who could still walk used a VR system at home for 8 weeks. Researchers m…

This study tested 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. Kids were split into three groups: VR training, biofeedback trainin…

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 doctors can accurately assess movement and quality of life in children with Duchenne muscular dystrophy through video calls instead of in-person visits. Twenty children aged 5-18 who can still walk took part. If remote assessments prove reliable, it c…

This study enrolled 150 people with Duchenne muscular dystrophy (DMD) to create a video library of their movement abilities at home. Caregivers recorded participants doing specific tasks using a secure app, and physical therapists scored the videos. The goal was to help validate …