Breakthrough injection stops devastating muscle disease before it starts
NCT ID NCT02386553
Summary
This study tested whether regular spinal fluid injections of an experimental drug called nusinersen could prevent or delay the onset of spinal muscular atrophy (SMA) in infants who had the genetic cause but showed no symptoms yet. It involved 25 infants under 6 weeks old who received the injections. The main goal was to see if the treatment could prevent the need for breathing support or death, and help infants reach normal movement milestones like sitting and walking.
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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, 60611-2605, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Columbia University
New York, New York, 10032, United States
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David Geffen School of Medicine
Los Angeles, California, 90095, United States
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Fondazione Serena Onlus - Centro Clinico Nemo
Milan, 20162, Italy
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Hacettepe University Medical Faculty
Ankara, 6230, Turkey (Türkiye)
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Hamad General Hospital
Doha, 3050, Qatar
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Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 807, Taiwan
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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National Taiwan University Hospital
Taipei, 100, Taiwan
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Nemours Children's Hospital, Orlando
Orlando, Florida, 32827, United States
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Ospedale Pediatrico Bambino Gesù
Rome, Lazio, 165, Italy
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Queensland Children's Hospital
South Brisbane, Queensland, 4101, Australia
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Royal Children's Hospital
Parkville, Victoria, 3052, Australia
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Seattle Children's Research Institute
Seattle, Washington, 98101, United States
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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The Johns Hopkins Hospital
Baltimore, Maryland, 21205, United States
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Universitaetsklinikum Freiburg
Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany
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University of California Davis Health System
Sacramento, California, 95817, United States
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University of Utah
Salt Lake City, Utah, 84112, United States
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Yeditepe University Medical School Hospital
Istanbul, 31755, Turkey (Türkiye)
Conditions
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