New hope for SMA: risdiplam shows promise in phase 2 trial
NCT ID NCT03032172
First seen Nov 01, 2025 · Last updated Apr 29, 2026 · Updated 21 times
Summary
This study looked at how safe and tolerable the drug risdiplam is for people with spinal muscular atrophy (SMA), a genetic disease that weakens muscles. It included 174 adults, children, and infants who had already tried other SMA treatments. Researchers monitored side effects and how the drug moved through the body, aiming to control the disease rather than cure it.
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This is a summary of
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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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Birmingham Heartlands Hospital
Birmingham, B9 5SS, United Kingdom
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Boston Childrens Hospital
Boston, Massachusetts, 02115, United States
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CHRU de Montpellier, Hopital Gui de Chauliac
Montpellier, 34295, France
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Columbia University Medical Center
New York, New York, 10032, United States
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Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Lombardy, 20122, Italy
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Hopital Femme Mere Enfant
Bron, 69677, France
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Hopital Roger Salengro
Lille, 59037, France
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Hopital des Enfants
Toulouse, 31059, France
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Hôpital Necker-Enfants Malades
Paris, 75015, France
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IRCCS Istituto Giannina Gaslini
Genoa, Liguria, 16147, Italy
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IRCCS Ospedale Pediatrico Bambino Gesù
Rome, Lazio, 00165, Italy
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Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie
Warsaw, 02-097, Poland
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Nemours Children's Hospital
Orlando, Florida, 32827, United States
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Policlinico Agostino Gemelli
Rome, Lazio, 00168, Italy
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Stanford University Medical Center
Palo Alto, California, 94304, United States
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The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, NE1 4LP, United Kingdom
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UCL Institute of Child Health & Great Ormond Street Hospital for Children
London, WC1N 1EH, United Kingdom
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UMC Utrecht
Utrecht, 3584 CX, Netherlands
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UOSD Malattie Neurodegenerative
Messina, Sicily, 98125, Italy
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UZ Gent
Ghent, 9000, Belgium
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UZ Leuven Gasthuisberg
Leuven, 3000, Belgium
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Universitäts-Kinderspital (UKBB) Neuropädiatrie
Basel, 4005, Switzerland
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Universitätsklinikum Essen
Essen, 45147, Germany
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Universitätsklinikum Freiburg
Freiburg im Breisgau, 79106, Germany
Conditions
Explore the condition pages connected to this study.