CBER is unanimously against Elevdiys’ return to the market without additional evidence, according to media reports citing an anonymous senior FDA official. Given Elevidys’ full approval, however, experts told BioSpace this path would set up a length legal battle between the regulator and

Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that have attracted attention from regulators.

Europe's medicines regulator did not recommend the approval of Sarepta Therapeutics' gene therapy Elevidys for a type of rare muscular dystrophy, the health agency said on Friday. Shares of the company were down nearly 17% at $10.

To suggest Sarepta Therapeutics may not survive its gene therapy crisis is a sobering claim to make, STAT's Adam Feuerstein writes. But it's an accurate reflection of the situation.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued the following statement:

Sarepta Therapeutics' licensing partner Arrowhead Pharmaceuticals said on Wednesday it expects to receive near-term milestone payments from the drugmaker despite recent setbacks, including the death of a trial patient reported last week.

This week on "The Readout LOUD" podcast, a mother whose son has Duchenne muscular dystrophy shares her perspective on Sarepta's gene therapy halt.