New drug combo aims to boost muscle power in SMA patients
NCT ID NCT05115110
First seen Nov 01, 2025 · Last updated Apr 28, 2026 · Updated 27 times
Summary
This study tests whether adding an experimental drug (RO7204239) that helps muscles grow to an existing SMA treatment (risdiplam) can improve motor function more than risdiplam alone. About 259 people with SMA, aged 2 to 25 years, will take part. The trial first finds the best dose, then measures changes in muscle strength and movement.
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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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Locations
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Asst Grande Ospedale Metropolitano Niguarda
Milan, Lombardy, 20162, Italy
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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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British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3N1, Canada
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CHULC, E.P.E. - Hospital Dona Estefania
Lisbon, 1169-045, Portugal
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Chr de La Citadelle
Liège, 4000, Belgium
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Clinical Hospital Centre Zagreb
Zagreb, 10000, Croatia
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Columbia University Medical Center
New York, New York, 10032, United States
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Fondazione IRCCS Istituto Neurologico ?Carlo Besta?
Milan, Lombardy, 20133, Italy
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Great Ormond Street Hospital For Children
London, WC1N 3JH, United Kingdom
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Hospital Sant Joan De Deu
Esplugues de Llobregas, Barcelona, 08950, Spain
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Hospital Universitario La Paz
Madrid, 28046, Spain
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Hospital Universitario la Fe
Valencia, 46026, Spain
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Hospital Vall d'Hebron
Barcelona, 08035, Spain
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Hospital de Santa Maria
Lisbon, 1649-035, Portugal
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IRCCS Istituto Giannina Gaslini
Genoa, Liguria, 16147, Italy
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Instytut Centrum Zdrowia Matki Polki
?ód?, 93-338, Poland
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Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, 04-730, Poland
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John Radcliffe Hospital
Oxford, OX3 9DU, United Kingdom
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Kagoshima University Hospital
Kagoshima, 890-8520, Japan
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Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie
Warsaw, 02-097, Poland
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Kobe University Hospital
Hyōgo, 650-0017, Japan
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McGill University Health Centre - Glen Site
Montreal, Quebec, H4A 3J1, Canada
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National Center for Global Health and Medicine
Tokyo, 162-0052, Japan
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Nemours Children's Hospital
Orlando, Florida, 32827, United States
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Neurology & Neuromuscular Care Center
Flower Mound, Texas, 75028, United States
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Ospedale Pediatrico Bambino Gesù
Rome, Lazio, 00165, Italy
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Ospedali Riuniti Torrette di Ancona
Ancona, The Marches, 60126, Italy
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Policlinico Agostino Gemelli
Rome, Lazio, 00168, Italy
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Sydney Children's Hospital
Randwick, New South Wales, 2031, Australia
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The Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
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UZ Gent
Ghent, 9000, Belgium
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Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX, Netherlands
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Uniwersytecki Szpital Kliniczny w Poznaniu
Późna, 60-355, Poland
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Uniwersyteckie Centrum Kliniczne
Gda?sk, 80-952, Poland
Conditions
Explore the condition pages connected to this study.