New hope for SMA patients: risdiplam shows promise in major trial
NCT ID NCT03032172
First seen Nov 01, 2025 · Last updated Jun 18, 2026 · Updated 31 times
Summary
This study tested a medicine called risdiplam in 174 adults and children with spinal muscular atrophy (SMA), a genetic disease that weakens muscles. The goal was to see if the drug is safe and how it works in the body. Participants had previously received other SMA treatments. The study was completed and focused on safety 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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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
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.