As it gears up to submit an approval application next year, Wave Life Sciences has presented fresh phase 2 data showing its ...

Shares of Sarepta Therapeutics Inc. have lost more than 26% this week, heading for their worst such performance in over a year, after the company disclosed the death of a Duchenne muscular dystrophy ...

Santhera's Duchenne muscular dystrophy (DMD) therapy Agamree has been cleared for NHS use after a confidential pricing agreement. The deal means that Agamree (vamorolone) will be available to ...