Business Wire

Destroy Duchenne selects DNA Nanobots as Partner to Develop Non-Viral Gene Delivery Solution for Duchenne Muscular Dystrophy

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) ...
Business Wire

Sarepta Announces First Clinical Data from siRNA Pipeline Targeting FSHD1 and DM1

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared the first clinical results from two of its siRNA ...
Benzinga.com

Why Is Rare Muscle Disease-Focused Dyne Therapeutics Stock Soaring Today?

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 ...
Phys.org

NanoCas, a smaller version of CRISPR tested with a single AAV, delivers on-target results

Mammoth Biosciences researchers have developed NanoCas, an ultracompact CRISPR nuclease, demonstrating its ability to perform gene editing in non-liver tissues, including skeletal muscle, using a ...
Morningstar

Dyno Therapeutics Unveils Best-in-Class Dyno-bn8 AAV Vector for Muscle Gene Therapies, Achieving Therapeutic Delivery With Improved Safety Potential

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

Dyne Therapeutics Announces Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients Demonstrating Promise of the FORCE™ Platform ...

- 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 ...
KXAN

Guidelines to standardize care, enhance safety monitoring, and promote equitable access to transformative treatments.

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 ...