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