Walking study for rare muscle and nerve conditions
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This study looks at how children and teens with Duchenne muscular dystrophy (DMD) or spinal muscular atrophy (SMA) walk, by having them do walking tests while wearing sensors. The goal is to find better ways to track how these diseases progress and how treatments work over time, without needing blood tests or scans.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a genetic test that confirms DMD or SMA (or be a healthy child without the condition).
- You must be able to walk on your own for at least 25 meters (about half a school bus length).
- For DMD: you must be taking corticosteroids (like prednisone) or have started them in the last 3 months.
- For SMA: you must be on a stable dose of an FDA-approved SMN therapy (like Spinraza or Evrysdi) for at least 6 months, or have received gene replacement therapy (Zolgensma).
- You cannot use braces, a walker, or a wheelchair to walk around outside your home.
- You cannot have had surgery or a recent injury (within 3 months) that affects how you walk.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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