New hope for SMA babies: risdiplam trial launches for newborns
NCT ID NCT05808764
First seen Nov 01, 2025 · Last updated May 18, 2026 · Updated 34 times
Summary
This study is testing a medicine called risdiplam in newborn babies with spinal muscular atrophy (SMA), a serious muscle-weakening disease. The goal is to see how the drug works in 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
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ann and Robert H. Lurie Children Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
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CHR Citadelle
RECRUITINGLiège, 4000, Belgium
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Children'S Hospital of Eastern Ontario
RECRUITINGOttawa, Ontario, K1H 8L1, Canada
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Clinic for Special Children.
RECRUITINGGordonville, Pennsylvania, 17529, United States
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Fondazione Policlinico Univeristario A. Gemelli
RECRUITINGROMA, Emilia-Romagna, 00168, Italy
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Fondazione Serena Onlus - CENTRO CLINICO NEMO
RECRUITINGMilano, Emilia-Romagna, 20162, Italy
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Hopital Universitaire des Enfants Reine Fabiola
RECRUITINGBrussels, 1020, Belgium
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Instytut Pomnik - Centrum Zdrowia Dziecka
RECRUITINGWarsaw, 04-730, Poland
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OUS (Oslo University Hospital), Rikshospitalet
RECRUITINGOslo, 0372, Norway
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UMC Utrecht
RECRUITINGUtrecht, 3508, Netherlands
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Universitatsklinikum Essen
RECRUITINGEssen, 45147, Germany
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University Of Michigan
RECRUITINGAnn Arbor, Michigan, 48109, United States
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Uniwersyteckie Centrum Kliniczne
RECRUITINGGdansk, 80-952, Poland
Conditions
Explore the condition pages connected to this study.