Gene therapy trial for fatal muscle disease halted after deaths
NCT ID NCT03199469
Summary
This study tested a one-time gene therapy called AT132 for young boys with X-linked myotubular myopathy (XLMTM), a rare and life-threatening muscle disease. The goal was to see if delivering a healthy gene could improve muscle function and reduce the need for breathing machines. The trial was stopped early because several children experienced severe complications, including deaths, after receiving the treatment.
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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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Hopital Armad Trousseau
Paris, 75012, France
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Hospital for Sick Children
Toronto, Ontario, M5G0A4, Canada
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Kinderklinik und Kinderpoliklinik im Dr. Von Haunerschen Kinderspital Klinikum der Universitat Munchen
München, 80337, Germany
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National Institute of Neurological Disorders and Stroke/NIH Porter
Bethesda, Maryland, 208892, United States
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UCLA Medical Center
Los Angeles, California, 90095, United States
Conditions
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