New hope for SMA babies: boosting gene therapy with a Follow-Up drug
NCT ID NCT07444450
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a drug called salanersen in babies with spinal muscular atrophy (SMA) who have already received gene therapy. The drug aims to help the body make more SMN protein, which is needed for muscle function. About 42 babies with two copies of the SMN2 gene will be randomly assigned to get either salanersen or a sham procedure. Researchers will track safety and motor milestones over up to 5.5 years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
salanersen (BIIB115)
What this could lead to
If it works, this could lead to better motor development and fewer symptoms for babies with SMA who have already had gene therapy.
What could go wrong
This is an early-stage trial with only 42 participants, so results may not apply to all. The sham procedure and lumbar puncture carry risks like discomfort or infection.
Disclaimer
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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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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.
Contacts and locations
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Study contacts
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