A research collaboration led by the Muscular Dystrophy Association (MDA) aims to create a molecular map of human muscle ...

Edgewise Therapeutics, Inc., , a leading muscle disease biopharmaceutical company, today announced that Behrad Derakhshan, Ph.D., Chief Operating Officer, will present at the Guggenheim Emerging ...

NMD Pharma is accelerating ignaseclant’s clinical development in CMT alongside multiple near-term clinical catalysts across its neuromuscular pipeline, with Phase 2 studies in spinal muscular atrophy ...

Muscular Dystrophy Association-led research collaboration with FSHD Society, LGMD2L Foundation, and Parent Project Muscular ...

A phase 2a trial of NMD Pharma’s rare disease prospect ignaseclant has missed its primary endpoint. | A phase 2a trial of NMD ...

The first-in-human clinical study of SRP-1005, known as INSIGHTT, is expected to begin in the second quarter of 2026CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the ...

Initiation of the IIMPACT study follows promising Phase 1 results where Restem-L demonstrated clinically meaningful efficacy ...

A group of local people who came together through Derry City and Strabane District Council’s GP Referral Scheme have formed ...

Discusses 3-Year Topline Results From EMBARK Phase 3 Trial of ELEVIDYS in Duchenne Muscular Dystrophy January 26, ...

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the 10 Cheap Stocks with Huge Upside Potential. On January 20, Canaccord Genuity raised BioMarin Pharmaceutical Inc.’s (NASDAQ:BMRN) price target ...