Vinay Prasad, a top regulator at the US Food and Drug Administration, has left the agency after a controversy over his ...

The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy ...

After our editorial, the agency relents to allow a Duchenne treatment.

While MSN reports that Sarepta’s stock price began to rise on Monday on the heels of the announcement, 5 the company is still facing challenges. Shipments of the gene therapy for non-ambulatory ...

Sarepta Therapeutics shares snapped a three-day losing streak on Tuesday, as analysts said resumption of U.S. shipments for ...

If drug developers are allowed to skip this step, it will mean that, in the near future, the first living body a new drug is ever tested in will be a human.

Ultragenyx’s 35% July selloff is overblown; both UX143 and UX111 programs remain on track with key catalysts ahead. Learn ...

This regulatory relief, combined with several other compelling catalysts, creates a pathway for the stock to potentially ...

In a statement, the Cambridge, Massachusetts-based company said that shipments of Elevidys (delandistrogene moxeparvovec) will restart while it continues dialogue with the FDA "on next steps in the ...

Sarepta Therapeutics (Nasdaq: SRPT) has been cleared by the American medicines regulator to resume shipments of its gene ...

For years, the Food and Drug Administration has been warning women about hormone therapies to treat menopause symptoms. The ...

Despite all the buzz around Sarepta and concerns about Elevidys, industry watchers aren’t convinced this current controversy ...