With a clutch of key data and planned regulatory applications this year from Avidity Biosciences, REGENXBIO and Capricor Therapeutics, CureDuchenne CSO Michael Kelly sees “momentum” in the Duchenne ...

Dosing has begun in a Phase 2 clinical trial testing the experimental oral therapy SAT-3247 in boys with Duchenne muscular ...

Durham-based Precision BioSciences, a gene-editing company, has received regulatory clearance to begin a clinical study of its potential treatment for Duchenne muscular dystrophy (DMD), a rare ...

Solid Biosciences Inc. (NASDAQ:SLDB) is entering a critical phase in its development of gene therapies for rare neuromuscular and cardiac diseases. The biotechnology company’s lead candidate, SGT-003 ...

A clinical trial testing Duchenne gene-editing therapy candidate PBGENE-DMD will soon launch in the U.S., having won ...

Precision BioSciences, Inc. , a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to ...

Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a favorable safety profile in young patients. Based on western blot testing, ...

– U.S. FDA Study May Proceed notification enables initiation of clinical trial site activation for the FUNCTION-DMD Phase 1/2 clinical study in patients with Duchenne muscular dystrophy (DMD) – – ...

DURHAM, N.C., July 23, 2025--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene ...

Feb 9 (Reuters) - The U.S. Food and Drug Administration on Thursday approved Marathon Pharmaceuticals' Duchenne muscular dystrophy (DMD) drug, Emflaza, to treat patients aged 5 years and above. DMD is ...

Sen. Roger Wicker urges states to add Duchenne muscular dystrophy to newborn screening, after federal health officials back ...