News

University of Louisville researchers have discovered that a mechanism involved in stem-cell renewal pathways and muscle tissue regeneration, with implications in muscular dystrophy research. The study, titled “TRAF6 regulates satellite stem cell self-renewal and function during regenerative myogenesis,” was published in the Journal of Clinical Investigation. In muscle injury a…

The effects of glucocorticoid therapy and bisphosphonate treatment on tissue structure and bone matrix mineralization of nine boys with Duchenne Muscular Dystrophy (DMD) was recently highlighted in a research paper entitled “Histomorphometry and Bone Matrix Mineralization Before and After Bisphosphonate Treatment in Boys with Duchenne Muscular Dystrophy: A Paired Trans-Iliac…

The Wnt signaling pathway might one day be a promising target for Duchenne muscular dystrophy (DMD) cognitive therapy, with potential clinical implications, according to a group of interdisciplinary researchers from universities throughout Chile and Australia. The team’s study, “Wnt signaling pathway improves central inhibitory synaptic transmission in a mouse model…

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…