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RGX-202, a gene therapy for Duchenne muscular dystrophy, was well tolerated with no therapy-related serious adverse effects in 3 patients who received the level 1 dosage.
Regenxbio Inc. announced that the FDA has granted fast track designation for RGX-202, a one-time gene therapy for treatment of Duchenne muscular dystrophy.According to a release from the company, the FDA previously granted orphan drug designation and rare pediatric disease designation for RGX-202, which is designed to support delivery and expression of genes in heart and skeletal muscle using the