Higher dose of SMA drug shows promise for those who switched treatments
NCT ID NCT05067790
First seen May 25, 2026 · Last updated May 25, 2026
Summary
This study looks at whether a higher dose of nusinersen can improve arm and hand function in teenagers and adults with spinal muscular atrophy (SMA) who cannot walk and have already taken another SMA drug called risdiplam. Participants receive two high loading doses, then regular maintenance doses over about 2 years, with an option to continue for another 2 years. The main goal is to measure changes in upper limb movement using a standard test.
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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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Barrow Neurological Institute
Phoenix, Arizona, 85013, United States
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Boston Children's Hospital
Boston, Massachusetts, 02115-5724, United States
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Columbia University
New York, New York, 10032, United States
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Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, 20133, Italy
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Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, 168, Italy
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Fondazione Serena Onlus - Centro Clinico Nemo
Milan, 20162, Italy
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Klinikum rechts der Isar der TU Muenchen
Munich, Bavaria, 81675, Germany
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Memorial Healthcare
Owosso, Michigan, 48867, United States
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NHO Osaka Toneyama Medical Center
Toyonaka-shi, Osaka, 560-8552, Japan
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Neurology Rare Disease Center
Denton, Texas, 76208, United States
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Ospedale S G Battista Molinette
Torino, 10126, Italy
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Rare Disease Research, LLC
Atlanta, Georgia, 30329, United States
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Semmelweis Egyetem
Budapest, 1085, Hungary
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Stanford Neuroscience Health Center
Palo Alto, California, 94304, United States
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The University of Texas Health Science Center at Houston
Houston, Texas, 77030, United States
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Universitaetsklinikum Essen
Essen, North Rhine-Westphalia, 45122, Germany
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Universitaetsklinikum Heidelberg
Heidelberg, Baden-Wurttemberg, 69120, Germany
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Universitaetsklinikum Ulm
Ulm, Baden-Wurttemberg, 89081, Germany
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University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
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University of Washington Medical Center
Seattle, Washington, 98195, United States
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Wake Forest University - School of Medicine - Central
Winston-Salem, North Carolina, 27157, United States
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Yokohama City University Hospital
Yokohama, Kanagawa, 236-0004, Japan
Conditions
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