New study tracks Risdiplam's Real-World impact on spinal muscle atrophy

NCT ID NCT07531719

Recruiting now Knowledge-focused Sponsor: Hoffmann-La Roche Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Aug 05, 2026 · Updated 4 times

Summary

This study will observe 30 children with type I or type II spinal muscle atrophy who are taking risdiplam. Researchers will track motor function improvements over 24 months using standard tests. The goal is to see how well the drug works in everyday medical practice.

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 young 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 not yet recruiting.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy spinal muscular atrophy, type 1 spinal muscular atrophy, type II

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

SPINAL MUSCLE ATROPHY

Contacts and locations

Locations

Air Force Specialized Hospital
RECRUITING

Cairo, DUMMY_VALUE, Egypt
Neurology Department, Ain Shams University Hospitals
RECRUITING

Cairo, 11566, Egypt

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