Early gene therapy trial for rare muscle disease halted
NCT ID NCT05906251
Summary
This early-stage study tested whether a gene therapy called SRP-6004 could safely deliver a working gene to people with limb girdle muscular dystrophy type 2B, a rare genetic muscle disorder. The trial involved just 2 participants who could still walk and had specific genetic mutations. Researchers gave the therapy through an IV infusion to see if it could help produce the missing muscle protein.
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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
Conditions
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