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...
What is Bobcat mRNA? How Does Bobcat mRNA Work for Duchenne Muscular Dystrophy?
Bobcat mRNA works differently than exon skipping or AAV-microdystrophy mechanisms. This next-generation method could lead to low-cost drugs for Duchenne muscular dystrophy.
Many exon skipping...
Next Generation Exon Skipping Therapies Developed for the Treatment of Duchenne Muscular Dystrophy
Next generation exon skipping therapies for Duchenne Muscular Dystrophy (DMD) represent a promising area of research aimed at addressing the underlying genetic mutations that...