Early treatment may help babies with spinal muscular atrophy sit without support
NCT ID NCT03779334
First seen Dec 11, 2025 · Last updated May 22, 2026 · Updated 22 times
Summary
This study tested a daily oral medicine called risdiplam in 26 infants who have a genetic diagnosis of spinal muscular atrophy (SMA) but do not yet show symptoms. The goal was to see if starting treatment early helps them reach motor milestones like sitting without support. Results showed that many infants achieved this milestone, suggesting early treatment may prevent or delay severe muscle weakness.
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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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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
Conditions
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