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

A new discovery about how tiny protein clusters form in cells could pave the way for treatments for Emery-Dreifuss muscular dystrophy (EDMD), a rare genetic disorder that causes muscle weakness and ...

ITF Therapeutics LLC, the U.S. affiliate of Italfarmaco, today announced the opening of the company's new U.S. headquarters in Concord, Massachusetts. The 10,000-square-foot facility located at 575 ...

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

FDA designations and new study publications Recent developments include Phase 2 DMD Study Breakthrough Results ...

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.

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

FDA grants Priority Review to Capricor's deramiocel for Duchenne cardiomyopathy, setting an August 2025 decision date with no ...

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

The Food and Drug Administration has accepted Capricor Therapeutics' application seeking approval of its cell therapy deramiocel for muscular dystrophy patients and granted it a priority review. The ...