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

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) ...

- 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 ...

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 ...

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

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 ...

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 ...

Muscle delivery: DYNE-251 showed a mean 657 ng/g PMO muscle drug concentration at 6 months. Exon 51 skipping: DYNE-251 demonstrated a mean absolute exon skipping level of 0.90% and a 0.80% change from ...