News

Early Duchenne MD Study Finds Drug Strengthens Musculature

Researchers have treated a zebrafish animal model with a tyrosine kinase inhibitor to study its effect on fish musculature and strength in an experiment that shows promise for future therapeutic targets to treat Duchenne Muscular Dystrophy (DMD). The study, entitled “Dasatinib as a treatment for Duchenne muscular dystrophy,” was published…

Duchenne MD Drug Candidate, Drisapersen, Still Lacking According to FDA Panel

A recent U.S. Food and Drug Administration (FDA) memorandum to the agency’s Peripheral and Central Nervous System Drugs Advisory Committee expresses skepticism regarding BioMarin Pharmaceutical Inc.’s Muscular Dystrophy drug candidate Kyndrisa (drisapersen) for treatment of Duchenne muscular dystrophy in patients with mutations amenable to exon 51 skipping. Duchenne muscular dystrophy…

DMD Trial of Sarepta Therapeutics’ Exon-skipping Drug Encouraging

A recent article published in the Annals of Neurology describes promising results from a Phase 2b study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). The study, titled “Longitudinal effect of eteplirsen vs. historical control on ambulation in DMD,” is the result of a collaborative effort between researchers from…

Muscular Dystrophy Therapy May Reside in Patient’s Own Cells

Muscular dystrophies (MDs) cause progressive weakness of muscles in young boys. Although treatments alleviate the symptoms and slow down disease progression, there is no cure for MDs. Research in regenerative medicine may offer novel therapies to restore muscle function in MDs patients as shown by scientists at KU Leuven University in…

Duchenne Muscular Dystrophy Also Involves Muscle Stem Cells

Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene expressed in differentiated myofibers. Researchers at The Ottawa Hospital and the University of Ottawa recently discovered that muscle stem cells also express dystrophin, affecting the generation of functional muscle fibers. Their discovery could revolutionize understanding of the disease and eventually…

Early Onset FSHD Patients Focus of New Clinical Study

aTyr Pharma, Inc., has announced an expansion of its Phase 1b/2 clinical trial to continue the development and evaluation of efficacy and safety of the Resolaris clinical program, for the treatment of early onset facioscapulohumeral muscular dystrophy (FSHD) patients. Facioscapulohumeral muscular dystrophy (FSHD) is a genetic muscular disorder, characterized by progressive muscle…