The desperate parents of a disabled child are pleading with the NHS to give their son access to a new drug. Jessica and ...

Patients with life-limiting condition set to receive new treatment at Children's Hospital but those at Heartlands must wait ...

Although only living in the Berkshires for a few years, Evan Butts has already ingrained himself into the community. A server ...

Sarepta Therapeutics Inc. misled investors about the safety and prospects for one of its Duchenne muscular dystrophy ...

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

Not too long ago, Editas Medicine was working on Reni-Cel, an investigational treatment for two rare blood disorders. Although the data from clinical trials for this medicine was promising, Editas ...

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 ...