Experimental gene therapy for rare muscle disease tested in small study
NCT ID NCT06747273
First seen Nov 01, 2025 · Last updated May 22, 2026 · Updated 24 times
Summary
This early-phase study tested a single dose of a gene therapy called SRP-9004 in 4 people with limb girdle muscular dystrophy type 2D/R3, a rare muscle-weakening disease. The main goal was to check safety, and researchers also looked at changes in muscle protein levels and function over time. The study was terminated 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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Children's Hospital of the King's Daughters
Norfolk, Virginia, 23510, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Conditions
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