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Gene Therapy

Duchenne Muscular Dystrophy Clinical Trials Muscular Dystrophy Mucopolysaccharidosis Type I RNA Interference Regulatory Affairs Antisense Oligonucleotides Nanomedicine

REGENXBIO Investors Face April 14 Deadline to Seek Lead Role in Securities Suit

Plaintiffs tie losses to FDA trial holds disclosed Jan. 28 after a reported tumor in a participant.

Dyne’s DMD Drug Hits Primary Endpoint in DELIVER REC, Shows Functional Gains

FDA Investigates Fatal Liver Failures Linked to Sarepta’s Elevidys Therapy