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 ...
Italfarmaco S.p.A. today announced a comprehensive update on the regulatory and clinical advancements for givinostat, 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.
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 ...
Pratteln, Switzerland, March 4, 2025 – Santhera Pharmaceuticals (SIX: SANN) today provides a trading update for the fiscal year 2024 and an outlook for 2025 and beyond.
MDH says early identification and treatment are crucial to slowing the progression of symptoms, as well as improving the quality of children’s lives.
MDH says early identification and treatment is key to slowing the progression of symptoms as well as extending and improving ...
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