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

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

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

The HHS said center directors deserve to be supported by managers aligned with aggressive goals “to expeditiously advance ...

The FDA said yesterday that it is investigating the deaths from liver failure of two non-ambulatory boys with Duchenne ...

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

Sarepta Therapeutics Inc. (NASDAQ:SRPT) is one of the best mid cap growth stocks to invest in now. Earlier on June 4, Sarepta ...

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

The death of a boy with Duchenne muscular dystrophy, tied to a gene therapy, is above all a tragedy. But it is also ...

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

Shares in both companies were down following news of the second death, casting fresh safety concerns over Elevidys.

FDA is investigating Sarepta's Elevidys after two deaths from liver failure in non-ambulatory DMD patients, raising safety ...