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 ...

Edgewise Therapeutics Inc. (NASDAQ:EWTX) saw its shares slide by 7.2% after the U.S. Food and Drug Administration (FDA) ...

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 ...

Investing.com -- Edgewise Therapeutics Inc (NASDAQ: EWTX) stock tumbled 7.2% after the company revealed that the U.S. Food and Drug Administration (FDA) deemed its CANYON trial data insufficient for ...

Writing in JAMA, four former governmental officials warn that the Trump administration’s involvement in delaying the approval ...

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).

Recent U.S. domestic news covers the FDA investigating deaths post-gene therapy by Sarepta, discussions at a CDC advisory ...