REGENXBIO Inc. provided an update on the RGX-314 programs, including the announcement that the pivotal program for RGX-314 for the treatment of wet age-related macular degeneration (wet AMD) is active.

Option Care Health Inc., the largest U.S. independent provider of home and alternate site infusion services, was selected to participate in the limited distribution network of VILTEPSO (viltolarsen) for patients with Duchenne Muscular Dystrophy who are amenable to exon 53 skipping therapy.

The U.S. Food and Drug Administration granted accelerated approval for NS Pharma’s Viltepso (viltolarsen) injection for patients with Duchenne muscular dystrophy who are amenable to exon 53 skipping therapy.

Great progress has been made in treating Duchenne muscular dystrophy in recent years, with many advancements via gene therapies. Rockville, Maryland-based ReveraGen BioPharma is taking a different approach.

The Food and Drug Administration accepted Sarepta Therapeutics Inc.’s New Drug Application seeking accelerated approval for golodirsen (SRP-4053), with a regulatory action date of Aug. 19, 2019.

U.S. health regulators granted priority review status for Sarepta Therapeutics Inc. SRPT -0.91 % ’s treatment of a rare genetic disorder. Sarepta is developing eteplirsen to treat Duchenne muscular dystrophy, […]