Gene therapy trial for rare muscle disease halted after severe side effects
NCT ID NCT03199469
First seen Nov 01, 2025 · Last updated May 13, 2026 · Updated 25 times
Summary
This study tested a gene therapy called AT132 for children under 5 with a rare, serious muscle disease (XLMTM) that causes breathing problems. The goal was to see if the therapy could reduce the need for breathing machines. However, the study was stopped early because some children had severe complications, including deaths. The 27 children who received the treatment are being followed for 10 years to monitor safety and muscle function.
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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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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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