DUCHENNE MUSCULAR DYSTROPHY (DMD)

This early-stage study tests the safety of a drug called rituximab in 5 boys aged 6 to 10 with Duchenne muscular dystrophy who can still walk. The drug works by lowering certain immune proteins to possibly slow muscle damage. Researchers will monitor side effects and check motor …

This study tests whether a special support group can improve the confidence (self-efficacy) of primary caregivers of children with spinal muscular atrophy or Duchenne muscular dystrophy. Thirty caregivers will take part and complete a questionnaire before and after the 8-week pro…

This study tests whether a gamified mobile app can help 46 boys aged 6–12 with Duchenne muscular dystrophy stay motivated with their physical therapy exercises. The app adds game elements like rewards and levels to a home exercise program. Researchers will measure physical functi…

This study looks at what influences how active children with Duchenne muscular dystrophy (DMD) are. Researchers will measure muscle strength, balance, thinking skills, sleep, and family factors in 30 children and their parents. The goal is to understand all the pieces that affect…

This study creates a registry (a detailed database) for people with Duchenne or Becker muscular dystrophy, including symptomatic female carriers. The goal is to collect information on health, quality of life, and how new treatments work in everyday care. Up to 1,500 participants …

This study looks at whether special watch-like devices worn on the ankles can measure how well toddlers with Duchenne muscular dystrophy (DMD) move during their daily activities. Researchers will ask 30 boys with DMD and 30 without (ages 1-3) to wear the devices for three 28-day …

This study looks at how the heart medication empagliflozin is processed in children with Duchenne muscular dystrophy (DMD) who have early heart damage. Researchers will give different doses to 10 children aged 8 to 18 and measure drug levels in their blood over 24 hours. The goal…