The company anticipates submitting a biologics licence application for accelerated approval in the US in early 2026.

Dyne anticipates filing a Biologics License Application (BLA) submission for US accelerated approval in early 2026.

Dire wolf pups born using gene-edited ancient DNA CRISPR used to match dire wolf genes in grey wolf embryos Dallas firm Colossal leads species restoration breakthrough ...

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD). 2 ...

The study revealed significant differences in multiple mtDNA deletions among the 3 groups. 6 TK2d is inherited in an autosomal-recessive pattern, meaning mutations must be present in both copies of ...

Get Instant Summarized Text (Gist) Gene therapy for Duchenne muscular dystrophy (DMD) faces challenges as the immune system may hinder its effectiveness. A large-scale trial showed that the ...

An independent data monitoring committee (DMC) favors continuing dosing Elevidys (delandistrogene moxeparvovec-rokl) to people with Duchenne muscular dystrophy (DMD) in ongoing clinical trials, ...

raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy (DMD), after the treatment failed to show significant benefit in a large-scale clinical trial.

Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 ...

The major reasons behind Duchenne muscular dystrophy include mutations that consist of internal deletions within the gene that codes for dystrophin, a protein that plays a critical role in the ...

Image credit: Shutterstock / Andrii Yalanskyi. Sarepta Therapeutics and Roche are looking to get approval to continue clinical trials of their Duchenne muscular dystrophy gene therapy Elevidys ...

Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a ...