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Malvern, Pennsylvania based PhaseBio Pharmaceuticals, Inc. , announced that the U.S. Food and Drug Administration (FDA) has granted the clinical-stage biopharmaceutical company orphan drug designation to Vasomera Injection for treating cardiomyopathy associated with dystrophinopathies — a spectrum of rare heterogeneous X-linked genetic muscle diseases caused by pathogenic variant mutations in the…

Marathon Pharmaceuticals has announced the launch of ACCESS DMD, its expanded access program (EAP) to provide deflazacort to qualified patients with Duchenne muscular dystrophy (DMD) under the authorization of the U.S. Food and Drug Administration (FDA). Marathon Pharmaceuticals, LLC, a biopharmaceutical company that develops treatments for rare neurological diseases,…

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…

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…

In a new study entitled “Jagged 1 Rescues the Duchenne Muscular Dystrophy Phenotype,” a team of researchers identified a single gene – Jagged1 – as the potential critical player capable of “rescuing” Duchenne muscular dystrophy phenotype in dogs that exhibit a complete absence of dystrophin. The study was published…

Results from a recent study published in the Journal of Pedriatric Orthopaedics showed full-time wheelchair use is a risk factor of first fracture among pediatric male patients with Duchenne or Becker muscular dystrophy. However, no correlation was found between fracture and corticosteroid use in these patient populations. Muscular…

Results from a study observing myotonic dystrophy type 2 (DM2) patients has helped pinpoint which disease symptoms are most important to patients with this rare type of muscular dystrophy. According to the study authors, the findings could help create a roadmap for doctors to prioritize treatments. The study titled “Patient-Reported…

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 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 (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…