Taiho Pharmaceutical Announced TAS-205 Phase 3 Clinical Study Did Not Meet Its Primary Endpoint
Taiho Pharmaceutical announced that the results of a phase III, randomized, placebo-controlled, double-blind and open-label, extension study of TAS-205 (Pizuglanstat) in patients with Duchenne...
Edgewise Therapeutics Reports Positive Results on Sevasemten Program for Becker and Duchenne Muscular Dystrophies
Edgewise Therapeutics, a leading muscle disease biopharmaceutical company, today unveiled positive results in its sevasemten program for Becker and Duchenne muscular dystrophies.
What is Sevasemten?Sevasemten:...
Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
Precision BioSciences, a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with...
Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy
As part of the FDA’s ongoing review, the Company has been informed that an Advisory Committee meeting is not indicated at this time. The...
Cumberland’s Ifetroban Demonstrated a 5.4% Improvement Compared to Placebo-Treated Patients
Cumberland Pharmaceuticals a specialty pharmaceutical company committed to developing new products for rare diseases, shared the latest positive results from its Phase 2 FIGHT...