A Novel “Mini-CRISPR” Could Be More Successful at Editing the Dystrophin Gene
Several research groups have been attempting to create compact CRISPR systems that can fit into a single AAV vector while maintaining editing capabilities. The...
Everything You Should Know About Genethon’s Gene Therapy GNT0004 for Duchenne Muscular Dystrophy
The pivotal phase of the trial, which is scheduled to begin in Q2 2025, is now being prepared by Genethon. What is Genethon’s Gene...
5 DMD Researches You Should Follow up in 2025
Despite significant advancements over the past ten years for DMD patients, there is still a significant unmet demand. In order to meet the need,...
Roche Announces New Results from the EMBARK Study of Elevidys in Outpatients with Duchenne Muscular Dystrophy (DMD)
Today, Roche revealed encouraging topline data from the second year of the EMBARK trial, a worldwide, double-blind, randomised phase III investigation of Elevidys (delandistrogene...
Personalized Miniorgans could Speed up Treatment of Duchenne
Personalized antisense oligonucleotides (ASOs) may accelerate studies on treating Duchenne muscular dystrophy (DMD).
Every year, tens of thousands of babies are born with enigmatic illnesses...