FDA is investigating Sarepta's Elevidys after two deaths from liver failure in non-ambulatory DMD patients, raising safety ...

The FDA said yesterday that it is investigating the deaths from liver failure of two non-ambulatory boys with Duchenne ...

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

Despite setbacks, Sarepta’s gene therapy pipeline and market leadership in DMD present a high-risk, high-reward setup. Read ...

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 ...

The death of a boy with Duchenne muscular dystrophy, tied to a gene therapy, is above all a tragedy. But it is also ...

The 12-month price targets, analyzed by analysts, offer insights with an average target of $72.34, a high estimate of $183.00 ...

Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene ...

Wall Street’s major market averages seesawed back and forth on Friday and finished mixed as Federal Reserve Governor ...

Sarepta Therapeutics Inc. (NASDAQ:SRPT) is a commercial-stage biopharmaceutical company that discovers and develops ...

Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic ...

The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...