Dyne Therapeutics Inc (NASDAQ:DYN) released initial clinical data from its ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 (DM1) and its DELIVER trial of DYNE-251 in patients with ...

Designation highlights significant unmet needs in DMD; dystrophin data from potentially registrational FORWARD-53 trial of WVE-N531 are on track for 3Q 2024 In previous Part A trial, WVE-N531 achieved ...

Dyne Therapeutics' early trial results for DM1 and DMD treatments show dose-dependent improvements, but data is preliminary. Despite promising DYNE-101 and DYNE-251 outcomes, limited participant ...

New York, Aug. 23, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) and Parent Project Muscular Dystrophy (PPMD) today announced the publication of jointly developed consensus ...

Mammoth Biosciences researchers have developed NanoCas, an ultracompact CRISPR nuclease, demonstrating its ability to perform ...

- All Evaluable Patients in the 3.4 mg/kg Cohort Treated with DYNE-101 Q4W Demonstrated Consistent Splicing Correction with a 19% Mean Improvement Across 22-Gene Panel at 3 Months - - Improvement in ...

Non-human primate study results demonstrate improvements in skeletal and cardiac muscle delivery efficacy, safety potential, human translatability, and manufacturability over prior AAV capsids ...

NORCO, Calif. & COLUMBUS, Ohio--(BUSINESS WIRE)--Destroy Duchenne, a nonprofit organization founded by DMD patient, Elijah Stacy that aims to Complete the Cure TM for Duchenne Muscular Dystrophy (DMD) ...

Scientists have developed a novel approach of genome editing to repair muscle stem (satellite) cells, offering new hope for Duchenne muscular dystrophy (DMD) treatment. While adeno-associated virus ...

The MarketWatch News Department was not involved in the creation of this content. -- Non-human primate study results demonstrate improvements in skeletal and cardiac muscle delivery efficacy, safety ...

The MarketWatch News Department was not involved in the creation of this content. Non-exclusive license for Solid Biosciences' proprietary, next-generation myotropic capsid, AAV-SLB101, to accelerate ...