Congress did not reauthorize the rare pediatric disease priority review program at the end of 2024. Advocates say the ripple effect is already being felt across biopharma.

Shares of Capricor Therapeutics (CAPR) closed Tuesday with modest gains and rose more than 3% in extended trading after the ...

Capricor Therapeutics, Inc.'s Deramiocel receives FDA Priority Review for DMD cardiomyopathy, with a decision by Aug. 2025.

The Food and Drug Administration (FDA) has granted Priority Review to the Biologics License Application (BLA) for deramiocel (CAP-1002) for the treatment ...

PepGen halts its Phase 2 CONNECT2 trial for PGN-EDO51 in DMD to assess 10 mg/kg cohort data, while advancing FREEDOM-DM1 studies in myotonic dystrophy.

PepGen is 'temporarily pausing' a Phase 2 study of its exon 51 skipping drug for Duchenne muscular dystrophy, it said Tuesday ...

On Tuesday, the FDA accepted for review Capricor Therapeutics Inc’s (NASDAQ:CAPR) Biologics License Application for ...

A Boston biotech's decision to pause a Duchenne muscular dystrophy study has resulted in a significant stock drop.

Capricor Therapeutics' shares rose after its biologics license application for deramiocel to treat a form of muscular dystrophy was given priority review by the Food and Drug Administration. Shares ...

While PepGen works to persuade the FDA to let it launch a phase 2 trial of its Duchenne muscular dystrophy (DMD) therapy in ...

In addition to the Priority Review, deramiocel has received Orphan Drug Designation from the FDA and EMA, as well as RMAT and ATMP Designations in the U.S. and Europe, respectively. Furthermore, ...

This is going to make a tremendous difference to his life’ - the words of doting uncle David Cook who helped to raise £28,000 ...