A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular ...

It's been nearly two years since Elevidys, a gene therapy for Duchenne MD developed by Sarepta Therapeutics, was approved by ...

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is ...

John Brandsema, MD, a pediatric neurologist at Children’s Hospital of Philadelphia, speaks with Healio about the latest ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

Despite being known for more than 150 years, Duchenne muscular dystrophy (DMD) remains an untreatable disease affecting approximately 1 of every 3,500-5,000 males. Muscles in patients express no or ...

A few weeks after Sarepta Therapeutics confirmed a patient died after receiving its drug Elevidys, research published April 3 ...

New Gene Therapy Approach Shows Promise for Duchenne Muscular Dystrophy July 24, 2024 — Researchers have made a significant breakthrough in developing a new gene therapy approach that restores ...

Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

DISCOVERY is a phase 1, proof-of-concept study evaluating safety and expression of the alpha-sarcoglycan protein after treatment with SRP-9004, an investigational gene therapy for the treatment of ...

Is dystrophin immunogenicity a barrier to advancing gene therapy for Duchenne muscular dystrophy? Gene Therapy , 2025; DOI: 10.1038/s41434-025-00531-y Cite This Page : ...