accelerated approval

Fierce Pharma

Sarepta, despite key trial flop, will seek full nod for DMD meds

Sarepta’s Vyondys 53 and Amondys 45 failed to show a statistically significant difference versus placebo on a key measurement of DMD patient mobility.

Angus Liu Nov 4, 2025 7:56am

Applied Tx's stock drops on non-update for rare disease asset

Sep 29, 2025 10:57am

Fierce Pharma

Stealth scores FDA approval for 1st Barth syndrome treatment

Sep 19, 2025 6:00pm

Daiichi, Merck rev up for accelerated approval filing

Sep 7, 2025 10:45am

Regenxbio's Hunter gene therapy latest to face FDA delay

Aug 19, 2025 10:30am

Cash-strapped Sangamo seeks partner for new gene therapy

Jun 24, 2025 11:53am