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.
A roundup of some of the latest scientific studies on the novel coronavirus and efforts to find treatments and vaccines for Covid-19, the illness caused by the virus.
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.
Vertex Pharmaceuticals will acquire privately held Exonics Therapeutics for an upfront payment of $245 million and potential milestone payments of up to $750 million.
Sarepta Therapeutics will buy privately held gene therapy company Myonexus Therapeutics for $165 million, moving deeper into an emerging and lucrative field within the biotech sector.
Sarepta Therapeutics’ shares plunged more than 40 percent after U.S. FDA staff reviewers maintained their negative outlook on the company’s rare muscle wasting disorder drug.
Biogen Inc. reported a better-than-expected quarterly profit as the company cut costs and demand rose for Tecfidera, its key drug for multiple sclerosis.