Controversy has dogged Sarepta’s Duchenne muscular dystrophy therapy Exondys 51 since its approval, and it’s not out of the woods yet. US cost-effectiveness watchdog the Institute for Clinical ...

The Sarepta and Biogen therapies mentioned are, respectively, Exondys 51 (eteplirsen) and Aduhelm (aducanumab). They are used for Duchenne muscular dystrophy and Alzheimer's disease. The FDA's ...

The US Food and Drug Administration approved Sarepta’s Exondys 51 (eteplirsen) injection, making it the first drug available for the rare genetic muscle-wasting disease in the country.

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...

Sarepta Therapeutics ... and other genetic therapeutic modalities for the treatment of rare diseases. It offers EXONDYS 51 injection to treat duchenne muscular dystrophy (duchenne) in patients ...

show flaws in the process US drug regulators use to speed approval for drugs to fill unmet needs, federal watchdogs said in a report. Biogen’s Aduhelm, Sarepta’s Exondys 51, along with Covis Pharma’s ...