Extra help for SMA kids: study tests boosting gene therapy results
NCT ID NCT04488133
Summary
This study looked at whether adding a drug called nusinersen could help young children with spinal muscular atrophy (SMA) who were still having movement problems even after receiving a one-time gene therapy. Researchers enrolled 46 children under age 3 to receive nusinersen injections into the spine over about two years. The main goal was to see if this extra treatment improved their ability to reach movement milestones like sitting, crawling, and standing.
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Contacts and locations
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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Arkansas Children's Hospital Research Institute
Little Rock, Arkansas, 72202, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital Philadelphia - Neurology
Philadelphia, Pennsylvania, 19104, United States
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Children's Hospital of The King's Daughters
Norfolk, Virginia, 23510, United States
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Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Milan, 20133, Italy
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Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, 00168, Italy
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Hospital Sant Joan de Déu
Esplugues Del Llobregat, Barcelona, 08950, Spain
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Hospital Universitario La paz
Madrid, 28046, Spain
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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Oregon Health and Science University (OHSU)
Portland, Oregon, 97239, United States
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Schneider Children's Medical Center
Petah Tikva, 4920235, Israel
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Stanford Neuromuscular Research
Palo Alto, California, 94304, United States
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Universitaetsklinikum Hamburg-Eppendorf
Hamburg, 20246, Germany
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University of Utah
Salt Lake City, Utah, 84112, United States
Conditions
Explore the condition pages connected to this study.