Experimental gene therapy tested for rare muscle disease
NCT ID NCT06747273
First seen Nov 01, 2025 · Last updated May 13, 2026 · Updated 23 times
Summary
This early-stage study tested a single dose of SRP-9004 gene therapy in 4 people with limb girdle muscular dystrophy type 2D/R3, a rare muscle-weakening disease. The main goal was to check safety, but researchers also looked at whether the therapy could increase a missing muscle protein. 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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