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Gene Therapy
Gene Therapy for Limb-Girdle Muscular Dystrophy
Recruiting1 awardPhase 1 & 2
Columbus, Ohio
This trial tests SRP-9003, a gene therapy, in patients with LGMD2E, a type of muscular dystrophy. The therapy uses a harmless virus to deliver a healthy gene into muscle cells to fix the genetic issue. SRP-9003 is a gene therapy that uses a harmless virus to deliver a healthy gene into muscle cells, a method previously demonstrated to be effective in treating muscular dystrophies.
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