Gene therapy trial for rare muscle disease halted after early phase
NCT ID NCT06747273
Summary
This early-stage study aimed to test the safety of a single-dose gene therapy called SRP-9004 for a rare, inherited muscle-wasting disorder. It involved a small group of participants, some who could walk and some who could not, to see if the treatment was safe and if it could increase a missing muscle protein. The trial was terminated, meaning it was stopped early, and only enrolled four people.
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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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