Sarepta Therapeutics (NasdaqGS:SRPT) recently provided updates that may have influenced its stock performance. The announcement of a safety update for ELEVIDYS, concerning severe side effects and a ...

Not too long ago, Editas Medicine was working on Reni-Cel, an investigational treatment for two rare blood disorders. Although the data from clinical trials for this medicine was promising, Editas ...

Writing in JAMA, four former government officials warn that the Trump administration’s involvement in delaying the approval ...

The agency is considering further regulatory action after a second Duchenne patient died from acute liver failure less than two months after receiving the gene therapy.