First human test of gene therapy for rare muscle disorder
NCT ID NCT03652259
Summary
This was an early-stage study to test the safety and initial effects of an experimental gene therapy called SRP-9003 for people with a rare, inherited muscle-wasting disease called LGMD2E. The therapy aimed to deliver a working copy of a faulty gene directly into the body via an IV infusion. The study was small, enrolling only 6 participants, and was later terminated.
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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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