Gene therapy trial offers hope for rare muscle disease
NCT ID NCT05230459
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a single dose of a gene therapy called AB-1003 in 10 adults with a rare genetic muscle disease (LGMD2I/R9). The goal is to see if it is safe and can help improve muscle function. Participants must be able to walk or run 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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Locations
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Kennedy Krieger Institute
RECRUITINGBaltimore, Maryland, 21205, United States
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Contact Email: •••••@•••••
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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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Contact Email: •••••@•••••
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VCU
RECRUITINGRichmond, Virginia, 23298, United States
Contact Email: •••••@•••••
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