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

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

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 ...

In one demonstration of potent therapeutic application, Scribe will present novel genome editing strategies using its X-Editor (XE) technology to target mutations in Duchenne Muscular Dystrophy ...

Duchenne muscular dystrophy (DMD), recognised by the ... affecting a significant number of affected individuals. Mutations in the DMD gene, which encodes the dystrophin protein, disrupt muscle ...

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 ...

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., ...

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 ...

Hopkins neuroscientist Richard Huganir is close to finding a potential life-changing treatment for kids with SYNGAP1-related ...

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