Participants with Duchenne muscular dystrophy (DMD) who received RGX-202 demonstrated functional improvement and better ...

The promise of genome editing to help understand human diseases and create new therapies is vast, but technological ...

The rationale and efficacy of each agent in pre-clinical or clinical studies are presented. Furthermore, a meta-analysis of gene profiling in DMD patients has been performed to understand the ...

Prime editing is an advanced gene editing technology that enables precise DNA corrections without introducing double-stranded ...

DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple ...

CureDuchenne is investing $1 million in Entos Pharmaceuticals to support the development of a new gene therapy for Duchenne muscular dystrophy (DMD) that aims to overcome the limitations of existing ...

ENTR-601-45 aims to correct the root cause of DMD by addressing mutations or absences in the DMD gene. The investigational therapy seeks to restore the mRNA reading frame, enabling the translation of ...

There are some approved gene therapies for DMD, including Roche and Sarepta’s Elevidys (delandistrogene moxeparvovec-rokl), which gained accelerated approval by the US Food and Drug Administration ...

INDIANAPOLIS (WISH) — It’s a devastating diagnosis that changes a child’s life forever, Duchenne muscular dystrophy, or DMD, is a rare and fatal genetic disease that primarily affects boys.

Genethon unveiled the 2-year follow-up data from its GNT0004 gene therapy clinical trial for Duchenne Muscular Dystrophy (GNT-016-MDYF) at the annual meeting of the American Society of Gene & Cell ...

PARIS--(BUSINESS WIRE)--Genethon unveiled the 2-year follow-up data from its GNT0004 gene therapy clinical trial for Duchenne Muscular Dystrophy (GNT-016-MDYF) at the annual meeting of the American ...

A personalized gene editing therapy was successfully used to treat an ... Casgevy was created using a CRISPR technology that directs the Cas9 enzyme to a specific location on DNA. Two new types of ...