Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...
This award honors an emerging leader shaping the future of neuromuscular research will be presented at the 2026 MDA Clinical ...
New York, Dec. 03, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today announced the awarding of seven new collaborative research grants totaling nearly $2 million to advance ...
A preclinical study led by the Germans Trias i Pujol Research Institute (IGTP), in collaboration with the Institut de ...
Myostatin, a protein that blocks muscle growth, has shown promising results as a potential therapeutic target for treating muscular dystrophy in animal studies, where its inhibition led to increased ...
– U.S. FDA has confirmed that screening and dosing may proceed in Study SRP-9005-101 for LGMD2C/R5 – Enrollment and dosing completed in Study SRP-9004-102 for LGMD2D/R3 – Data expected for SRP-9003 ...
Multiple biological pathways go awry to cause disease. Cambridge startup Catabasis is developing a platform to simultaneously modulate multiple targets in a disease pathway, with a focus on diseases ...
The muscle destruction associated with Duchenne muscular dystrophy (DMD), the most common childhood form of muscular dystrophy, is halted in mice when supplemental amounts of a naturally occurring ...
PTC Therapeutics withdrew a New Drug Application for Translarna, a muscular dystrophy treatment, after the Food and Drug Administration indicated it would likely not grant approval.