Duchenne muscular dystrophy is characterized by a mutation in the DMD gene that leads to a lack of dystrophin and muscle loss ...

Elevidys has been given full approval to treat ambulatory patients with DMD, with an accelerated approval in non-ambulatory ...

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

The FDA is investigating two deaths in non-ambulatory Duchenne muscular dystrophy patients who received a Sarepta gene ...

The first procedure in the world designed to treat a patient using a bioengineered external liver was recently performed by a team at Intermountain Medical Center.

The FDA is assessing the need for “further regulatory action” on Sarepta's Duchenne muscular dystrophy gene therapy in the ...

The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...

United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, and Intermountain Health, one of the nation’s top health systems and the largest nonprofit health system in the ...

The commercial and clinical use of Elevidys was discontinued after two fatal cases of acute liver failure.

The only option left for the doctors was liver transplantation and Anakendu's mother came to her rescue by volunteering to ...