Through the program, gene therapies are developed to treat patients with retinitis pigmentosa caused by pathogenic variants ...

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

"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, ...

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

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

Capricor Therapeutics has received an orphan drug designation for its drug aimed at potentially treating Becker Muscular Dystrophy. The biotechnology company said Tuesday that the Food and Drug ...

Access free, up-to-date clinical references regarding differential diagnosis of age-related macular degeneration. Healio's Clinical Guidance content provides valuable insights for physicians.

The Scientist's content tagged with: Stargardt's Macular Dystrophy. Login. Subscribe. News; Magazines. An Interactive Experience. Current Issue. April 1, 2025. View this Issue. Archive. Quarterly ...

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

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 ...

SANTIAGO, Chile (AP) — As a child, Susana Moreira didn’t have the same energy as her siblings. Over time, her legs stopped walking and she lost the ability to bathe and take care of herself ...

Over the last two decades, the 41-year-old Chilean has spent her days bedridden, suffering from degenerative muscular dystrophy. When she finally loses her ability to speak or her lungs fail, ...