Keyword: Duchenne muscular dystrophy (DMD)

Sarepta will negotiate a warning for golodirsen rather than carry out more studies. But the implications of the rejection range beyond golodirsen.

Pfizer halted two phase 2 trials of its Duchenne muscular dystrophy drug domagrozumab after the main efficacy study failed its primary endpoint.

Raxone is currently in a phase 3 study on DMD patients who are taking glucocorticoids.

Sarepta Therapeutics has penned its second DMD gene therapy pact this year.

Ed Kaye, who did what looked impossible a year ago and got Sarepta’s controversial Duchenne med approved, will step down later this year.

As it continues its sales push for the controversial Duchenne med Exondys 51 (eteplirsen), Sarepta has taken on a new chief medical officer to help it develop its next-gen DMD pipeline.

Sarepta added more than 75% in early trading on news that its Duchenne muscular dystrophy (DMD) treatment Exondys 51 has been approved.

The FDA notified Santhera Pharmaceuticals that it would require the results from another trial, which is expected to take an additional three years.