Experimental gene therapy for rare muscular dystrophy shows early promise but study halted
NCT ID NCT03652259
First seen Nov 01, 2025 · Last updated May 13, 2026 · Updated 26 times
Summary
This study tested a gene therapy called SRP-9003 for people with limb-girdle muscular dystrophy type 2E, a rare genetic muscle-weakening disease. Six participants received a one-time infusion to deliver a working copy of the missing gene. The study was terminated early, so final results are limited, but researchers looked at safety and whether the therapy could increase protein levels in muscle.
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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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Locations
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Conditions
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