Gene therapy hope for rare muscular dystrophy
NCT ID NCT05876780
First seen Mar 06, 2026 · Last updated May 16, 2026 · Updated 13 times
Summary
This early-stage study tests a single-dose gene therapy called SRP-9003 in 6 people with limb girdle muscular dystrophy type 2E/R4, a rare genetic muscle-weakening disease. The main goals are to check safety and see if the therapy can restore a missing muscle protein. Both walkers and non-walkers can join.
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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
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St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
Conditions
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