Upcoming Exon 44 Skipping Therapies for the Treatment of Duchenne Muscular Dystrophy
Exon 44 skipping therapies represent a promising area in the treatment of Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle weakness...
Russian Biotech Company Generium has Begun Clinical Trials of a Gene Therapy for Duchenne Muscular Dystrophy
Clinical studies for a therapy for Duchenne muscular dystrophy have begun at Sirius University and the Russian biotech business Generium. At a gathering at...
Cumberland Pharmaceuticals Announces Fantastic Results from Phase 2 FIGHT Duchenne Trial in DMD Heart Disease
Cumberland Pharmaceuticals, a specialty pharmaceutical company with development efforts focused on new products for rare diseases, today announced positive top-line results from its Phase...
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...