New hope for SMA babies: risdiplam trial launches in newborns
NCT ID NCT05808764
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study is testing the drug risdiplam in newborn babies with spinal muscular atrophy (SMA), a serious muscle-weakening disease. The goal is to see how the drug moves through the body and if it is safe for infants under 20 days old. Ten babies will take part, and researchers will monitor side effects and drug levels in the blood.
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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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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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Locations
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Ann and Robert H. Lurie Children Hospital of Chicago
Chicago, Illinois, 60611, United States
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CHR Citadelle
Liège, 4000, Belgium
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Children'S Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1, Canada
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Clinic for Special Children.
Gordonville, Pennsylvania, 17529, United States
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Fondazione Policlinico Univeristario A. Gemelli
ROMA, Emilia-Romagna, 00168, Italy
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Fondazione Serena Onlus - CENTRO CLINICO NEMO
Milano, Emilia-Romagna, 20162, Italy
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Hopital Universitaire des Enfants Reine Fabiola
Brussels, 1020, Belgium
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Instytut Pomnik - Centrum Zdrowia Dziecka
Warsaw, 04-730, Poland
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OUS (Oslo University Hospital), Rikshospitalet
Oslo, 0372, Norway
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UMC Utrecht
Utrecht, 3508, Netherlands
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Universitatsklinikum Essen
Essen, 45147, Germany
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University Of Michigan
Ann Arbor, Michigan, 48109, United States
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Uniwersyteckie Centrum Kliniczne
Gdansk, 80-952, Poland