Repurposing existing therapies may help slow muscle weakness in patients with Duchenne muscular dystrophy, a new study ...

Phil Lambert, Ph.D., chief scientific officer of Satellos Bioscience Inc., explains the mechanism of action of the company's experimental Duchenne muscular dystrophy drug, SAT-3247, and the thinking ...

Pepgen will stop development of its Duchenne MD exon-skipping therapy PGN-ED051 after increases in dystrophin levels proved ...

Dystrophin's isoforms are thought to contribute variably to neuropsychiatric disorders, behavioral disorders, and ...

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the ...

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...

Entrada has secured the authorisation within the EU Clinical Trial Regulation to begin the Phase I/II trial of ENTR-601-45.

CureDuchenne Ventures announced an initial $1M investment in Entos Pharmaceuticals Inc (Entos), a biotech company that develops genetic medicines utilizing its non-viral, redosable Fusogenix PLV ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following ...

Multiple first-in-human trials showed functional and biomarker improvements in early data reported at ASGCT's annual meeting.

clinical study to evaluate Entrada’s investigational medicinal product ENTR-601-45 for the potential treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation in the ...

An abnormality in the DMD gene causes Duchenne muscular dystrophy. The DMD gene produces dystrophin, a protein that maintains the structure of muscle cells. People with DMD have mutations (changes ...