MILAN--(BUSINESS WIRE)--Italfarmaco Group announced today topline data from its proof-of-concept Phase 2 trial with Givinostat, the company’s proprietary histone deacetylase (HDAC) inhibitor, in 51 ...

SAN DIEGO, June 17, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare ...

BOULDER, Colo.--(BUSINESS WIRE)--Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the company will present data on sevasemten, an ...

NEWPORT BEACH, Calif., May 21, 2025 /PRNewswire/ -- CureDuchenne, a global nonprofit committed to finding and funding a cure for Duchenne muscular dystrophy, proudly announces the appointment of ...

Parent Project Muscular Dystrophy (PPMD), in partnership with the Foundation to Eradicate Duchenne (FED), today announced Representative Troy Balderson (OH-12), Senator Susan Collins (ME), Senator Amy ...

The FDA has granted Orphan Drug designation to EB 002 ((+)-epicatechin; Epirium Bio) for the treatment of Duchenne and Becker muscular dystrophy. The Food and Drug Administration (FDA) has granted ...

When you think about children diagnosed with Duchenne and Becker muscular dystrophies, the first things that usually come to mind are difficulty walking and the progressive skeletal muscle wasting, ...

Our choice of psychological adaptation to DBMD as the study outcome is consistent with the preferences expressed during community engagement (described below). Predictor variables were chosen based on ...

For genetic conditions like Duchenne muscular dystrophy, there is little doctors can do to slow or treat the condition other than trying to manage symptoms, since only addressing the genetic changes ...

U.S. FDA grants Orphan Drug Designation to Deramiocel for the treatment of Becker Muscular Dystrophy, broadening Capricor’s focus in neuromuscular diseases Capricor remains on track for the August 31, ...

– Company to host an Industry Forum to discuss the lived experience of Becker and clinical advancements featuring a leading neuromuscular disease expert and a patient advocate – Edgewise is sponsoring ...