FDA has indicated that an Advisory Committee meeting is not required at this timeIn-person late-cycle review meeting ...

"We just try to take it one day at a time and do the best we can and enjoy every day," said 16-year-old Brandon's mother, ...

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

Capricor Therapeutics updates on Deramiocel BLA for duchenne muscular dystrophy: San Diego Thursday, June 26, 2025, 15:30 Hrs [IST] Capricor Therapeutics, a biotechnology company ...

Three conditions that likely won’t qualify under the Department for Work and Pensions' new ‘severe conditions criteria’ have ...

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

Edgewise Therapeutics, Inc., a leading muscle disease biopharmaceutical company, unveiled positive results in its sevasemten programme for Becker and Duchenne muscular dystrophies. The company ...

Muscular Dystrophy is a degenerative disease that is particularly common in young boys and men. Joey, known for his "Canuck ...

Capricor's DMD therapy Deramiocel is under FDA Priority Review with no major issues flagged; PDUFA date set for August 31, 2025.

Capricor Therapeutics said on Tuesday the U.S. Food and Drug Administration will not convene its panel of outside experts before deciding on the company's cell therapy for a heart condition associated ...

Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage ophthalmic biopharmaceutical company developing gene therapies for the ...