The FDA found that data from a single Phase II study were “insufficient” to justify an accelerated approval review for ...

Edgewise reports sustained functional stability in Becker patients and selects Phase 3 dose for Duchenne after positive Phase ...

Edgewise Therapeutics (NASDAQ:EWTX) lost ~9% in the morning hours on Thursday after the company posted data from clinical ...

The agency is considering further regulatory action after a second Duchenne patient died from acute liver failure less than two months after receiving the gene therapy.

New open label data in Becker demonstrated sustained disease stabilization up to three years, reinforcing prior clinical findings – – Ongoing pivotal trial and FDA Type C meeting provide clear path to ...

Edgewise Therapeutics Inc. CEO Kevin Koch speculated that “perhaps a different environment at the FDA” from four months ago ...

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

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

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

Precision BioSciences, Inc. received a Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for PBGENE-DMD to treat Duchenne muscular dystrophy (DMD).

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 ...