First human test of gene therapy for rare muscle disease
NCT ID NCT05876780
Summary
This early-stage study is testing a single dose of an experimental gene therapy called SRP-9003 in six people with a rare genetic muscle disease called limb girdle muscular dystrophy type 2E/R4. The main goals are to check if the treatment is safe and to see if it increases a needed muscle protein. Researchers will measure changes in muscle strength and movement ability over five years.
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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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