Gene therapy trial opens for rare Muscle-Wasting condition
NCT ID NCT05230459
First seen Oct 31, 2025 · Last updated May 19, 2026 · Updated 32 times
Summary
This study tests a single dose of a gene therapy called AB-1003 in 10 adults with limb girdle muscular dystrophy type 2I/R9, a genetic condition that weakens muscles. The main goal is to check safety and find the right dose. Participants must be able to walk 10 meters in under 30 seconds.
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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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Kennedy Krieger Institute
RECRUITINGBaltimore, Maryland, 21205, United States
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University of California - Irvine
RECRUITINGIrvine, California, 92697, United States
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University of Iowa
RECRUITINGIowa City, Iowa, 52242, United States
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University of Kansas Medical Center
RECRUITINGKansas City, Kansas, 66160, United States
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University of Washington Medical Center
RECRUITINGSeattle, Washington, 98195, United States
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VCU
RECRUITINGRichmond, Virginia, 23298, United States
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Conditions
Explore the condition pages connected to this study.