New drug shows promise in preventing SMA in newborns
NCT ID NCT03779334
First seen Dec 11, 2025 · Last updated Jun 23, 2026 · Updated 27 times
Summary
This study tests the drug risdiplam (Evrysdi) in infants up to 6 weeks old who have a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. The goal is to see if early treatment can help them reach motor milestones like sitting without support. The trial involves 26 participants and is currently active but not recruiting.
Disclaimer
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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.
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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Locations
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Chr de La Citadelle
Liège, 4000, Belgium
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Hospital das Clinicas - FMUSP_X
São Paulo, São Paulo, 05403-000, Brazil
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Kaohsiung Medical University Chung-Ho Hospital
Kaohsiung City, 807, Taiwan
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Nemours Children's Hospital
Orlando, Florida, 32837, United States
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Russian Children Neuromuscular Center of Veltischev
Moscow, Moscow Oblast, 125412, Russia
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Sydney Children's Hospital
Randwick, New South Wales, 2031, Australia
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Szpital Gdanskiego Uniwersytetu Medycznego
Gda?sk, 80-952, Poland
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
risdiplam (Evrysdi)
What this could lead to
If successful, this could prevent or delay the onset of spinal muscular atrophy in infants diagnosed before symptoms appear, allowing them to achieve normal motor milestones.
What could go wrong
This is a small Phase 2 study with only 26 infants, so results may not apply to all patients. Long-term safety and efficacy are still being evaluated, and some children may still develop symptoms or need ongoing treatment.
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.