Experimental gene therapy for rare muscle disease shows early promise but trial stopped
NCT ID NCT03652259
First seen Nov 01, 2025 · Last updated May 18, 2026 · Updated 27 times
Summary
This early-stage study tested a gene therapy called SRP-9003 in 6 people with Limb-Girdle Muscular Dystrophy Type 2E, a rare genetic condition that causes muscle weakness. The goal was to see if the treatment was safe and could help muscles work better. The study was stopped early, so results are limited.
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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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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Conditions
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