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

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 ...

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

Sarepta Therapeutics, Inc. , the leader in precision genetic medicine for rare diseases, today announced that Medsafe, the New Zealand Medicines and Medical Devices Safety Authority, has granted ...

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