New study to track how risdiplam helps kids with spinal muscle atrophy in daily life
NCT ID NCT07531719
First seen Apr 24, 2026 · Last updated Jun 20, 2026 · Updated 13 times
Summary
This study will follow 30 children with Type I or Type II spinal muscle atrophy who are taking risdiplam. Researchers will track their motor skills and safety over 24 months using standard tests. The goal is to see how well the drug works in real-world settings, not just in controlled trials.
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
risdiplam
What this could lead to
If successful, this study could provide real-world evidence on how well risdiplam improves motor function in children with spinal muscle atrophy.
What could go wrong
This is an observational study, not a controlled trial, so results may be less definitive. It is also small (30 participants) and has not yet started recruiting.
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.