FDA says no to Sarepta's golodirsen, blocking bid to expand ...

ICER posts damning report on Sarepta’s Duchenne drug Exondys...

Controversy has dogged Sarepta’s Duchenne muscular dystrophy therapy Exondys 51 since its approval, and it’s not out of the woods yet.

Sarepta DMD gene therapy could improve patients' motor funct...

Sarepta Therapeutics announced potentially game-changing results from its Duchenne muscular dystrophy gene therapy candidate, with a small phase 1 trial showing an improvement in patients’

Sarepta dives deeper into gene therapy

Sarepta Therapeutics has handed $30 million to Lacerta in exchange for a group of gene therapies, including a Pompe disease candidate.

Summit shares plunge nearly 80% after DMD drug fails

Summit Therapeutics has axed development of its Duchenne muscular dystrophy drug ezutromid after a spectacular failure in phase 2, causing the UK biotech's shares to plunge.

Pharma collaborates to improve chances of NICE backing Duche...

Project will initially focus on DMD but could benefit other rare diseases