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.
Researchers at Technical University of Munich (TUM) recently developed a gene therapy that might cure Duchenne muscular dystrophy.
Shares of Sarepta Therapeutics Inc. soared 32 percent after U.S. regulators shocked Wall Street by reversing their rejection of the muscle-wasting disorder therapy less than four months earlier.
Shares in Solid Biosciences Inc. plunged 71 percent after a clinical trial testing the company’s gene therapy for a muscle-wasting disorder was halted for the second time in less than two years.
Shares of Sarepta Therapeutics Inc. plunged 14 percent after the U.S. health regulator, in an unexpected move, declined to approve the drugmaker’s newest treatment for a muscle-wasting disorder.