StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.
When Mats died of a terminal disease, messages flooded to his parents from the community of gamers with whom he had an unknown, vibrant life. Good luck keeping your eyes dry.
Investors consider Dyne Therapeutics' treatments for muscle diseases and strong cash position as a speculative Buy ...
CHARLESTOWN, Mass., Nov. 06, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the "Company” or "Solid”), a ...
WALTHAM, Mass., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for ...
CPVT is an underdiagnosed and highly malignant, genetic arrhythmia syndrome with historic mortality rates reaching as high as 50% by age 35.1 The treatment landscape has not meaningfully changed in ...
Dyne reported positive efficacy data and a favorable safety profile1 from the ongoing Phase 1/2 global DELIVER trial of DYNE-251 in males with Duchenne muscular dystrophy (DMD) mutations amenable ...
Good morning, and welcome to the Wave Life Sciences Third Quarter 2024 Financial Results Conference Call. At this time, all participants are in a listen only mode. As a reminder, this call is being ...
We have commenced the submission of our BLA which we expect to be complete by year end and we have significantly strengthened our balance sheet in order to scale ... development for the treatment of ...