Research

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...

Next Generation Lipid Nanoparticles (LNPs) for the Treatment of Duchenne

Delivering the dystrophin protein to the afflicted muscles is one of the most difficult problems in gene therapy for DMD. It may be possible...

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