Business Insider

Muscular Dystrophy Association Announces nearly $2 Million in Collaborative Research Grants Advancing Treatments Across Multiple Neuromuscular Diseases

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 ...
Nasdaq

Capricor Therapeutics Receives Orphan Drug Designation for Deramiocel in Becker Muscular Dystrophy, Expanding Focus in Neuromuscular Diseases

Capricor Therapeutics announced that the U.S. FDA has granted Orphan Drug Designation to its lead cell therapy candidate, Deramiocel, for the treatment of Becker Muscular Dystrophy (BMD). This ...
Nasdaq

Cumberland Pharmaceuticals Announces Breakthrough Results from the Phase 2 FIGHT DMD Trial in Duchenne Muscular Dystrophy Heart Disease

NASHVILLE, Tenn., Feb. 4, 2025 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, ...
EurekAlert!

Genethon signs licensing agreement with AskBio for the development of an investigational gene therapy for Pompe disease

Genethon, a leading laboratory in gene therapy for rare diseases, today announced that it has entered into an exclusive, worldwide licensing agreement with AskBio, a gene therapy company wholly owned ...