CureDuchenne is investing $1 million in Entos Pharmaceuticals to support the development of a new DMD gene therapy.

Months after causing safety concerns that led the FDA to hit a related trial with a clinical hold, a phase 2 trial of ...

A rare and fatal form of muscular dystrophy has long posed a devastating diagnosis for children, primarily affecting boys.

DMD treatment SAT-3247 was safe and well-tolerated, and showed signs of increasing muscle strength in five adults, Satellos ...

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

Local intramuscular administration of the antisense oligonucleotide PRO051 in patients with Duchenne's muscular dystrophy with relevant mutations was previously reported to induce the skipping of ...

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

Sarepta Therapeutics, Inc. , the leader in precision genetic medicine for rare diseases, today reported new results from Study 9001-103. Also known as ENDEAVOR, Study 9001-103 is a multi-cohort study ...

Japanese MHLW approves Chugai’s Elevidys as a gene therapy product to treat Duchenne muscular dystrophy: Tokyo Thursday, May 15, 2025, 16:00 Hrs [IST] Chugai Pharmaceutical Co., ...

adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene ...

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 ...