DUCHENNE MUSCULAR DYSTROPHY (DMD)

This study aims to check if a drug called rituximab is safe for children with Duchenne muscular dystrophy (DMD) who can still walk. It will involve 5 boys aged 6-10 and follow them for one year, closely watching for any side effects. The researchers will also measure changes in t…

This study is testing whether a mobile app that turns physical therapy exercises into a game can help boys with Duchenne muscular dystrophy (DMD) stay motivated and stick to their home exercise routines. The goal is to see if this fun, digital approach helps them maintain their p…

This study aims to see if an 8-week online support group can help improve the confidence and coping skills of parents and caregivers looking after children with severe muscle diseases (SMA or DMD). Researchers will work with 30 caregivers in Pakistan, providing them with group se…

This study aims to understand how a heart medication called empagliflozin is processed by the bodies of children with Duchenne muscular dystrophy (DMD). Researchers will give the drug to 10 young DMD patients of different ages and sizes and measure its levels in their blood over …

This study aims to understand how the use of home mechanical breathing machines has changed in Denmark over the past 10 years. Researchers will look at data from about 450 patients with conditions like ALS or spinal cord injuries who needed this type of long-term breathing suppor…

This study aims to understand how Duchenne muscular dystrophy (DMD) affects babies and toddlers in their first three years of life. Researchers will follow 105 boys with DMD, tracking their motor skills, language development, and when they start treatments. The goal is to gather …

This study is creating a patient registry to collect health information from people with Duchenne or Becker muscular dystrophy. It will track how new treatments work in real-world settings and aim to improve care quality across German-speaking countries. The registry will include…

This study aims to see if a special watch-like device worn on the ankles can reliably measure walking ability in very young boys (aged 1-3) with Duchenne muscular dystrophy (DMD). Currently, there's no good way to test how well potential new medicines work in toddlers. The study …

This study aims to understand what helps or hinders physical activity in children with Duchenne muscular dystrophy (DMD). Researchers will examine 30 children with DMD and their parents, looking at factors like muscle function, balance, sleep, behavior, and family support. The go…