Italfarmaco S.p.A. today announced a comprehensive update on the regulatory and clinical advancements for givinostat, the ...

Researchers, physicians, families and affected patients view Duchenne muscular dystrophy (DMD) as a neuromuscular condition. Children are diagnosed during the first years of life because they have ...

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

PepGen has previously faced an FDA clinical hold on the investigational new drug application of PGN-ENO51 in the Phase II ...

Duchenne muscular dystrophy causes progressive muscle degeneration and weakness, but physical exercise should still be part of a comprehensive treatment strategy. Duchenne muscular dystrophy (DMD ...

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.

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

Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is ...

Medically reviewed by Smita Patel, DO Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed ...

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

There is a broad consensus in the medical community in support of Duchenne newborn screening and we in Connecticut are hoping ...