New drug shows promise in preventing SMA symptoms in babies
NCT ID NCT03779334
First seen Dec 11, 2025 · Last updated May 15, 2026 · Updated 21 times
Summary
This study tests an oral medicine called risdiplam 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 helps them reach motor milestones like sitting without support. 26 infants took part, and the study measures how many can sit independently and avoid severe breathing problems or death.
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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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