Can gene therapy help babies with SMA reach milestones? a Real-World review
NCT ID NCT07737756
First seen Jul 30, 2026 · Last updated Jul 31, 2026 · Updated 1 time
Summary
This study looks back at medical records of children with spinal muscular atrophy (SMA) type 1 who received a one-time gene therapy called onasemnogene abeparvovec (Zolgensma). Researchers want to see if treated infants can sit independently for 30 seconds or more within a year. The study includes only 6 patients from one center in Saudi Arabia, so it offers a small, real-world snapshot of how the therapy works outside of formal trials.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- a gene therapy called onasemnogene abeparvovec (Zolgensma)
- What this could lead to
- If successful, this study could provide real-world evidence on how well gene therapy works for SMA type 1 outside of clinical trials.
- What could go wrong
- This is a small, retrospective chart review of only 6 patients at a single center, so results may not apply broadly. It looks back at existing records, not a controlled experiment.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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