News

Novel Duchenne MD Drug Delayed by Additional FDA Requirements

A novel RNA-based drug from specialized biotech company, Sarepta Therapeutics, Inc., is experiencing a delay in its New Drug Application (NDA) from the US Food and Drug Administration’s (FDA), due to the FDA’s request for specific, additional data requirements from the company. The drug, eteplirsen, is one of the company’s leading pipeline…

Translarna Posts Encouraging Phase 2b Data For Treating nmDMD

PTC Therapeutics, Inc. announced the results of a Phase 2b clinical trial entitled “Ataluren treatment of patients with nonsense mutation dystrophinopathy” in the October issue of Muscle & Nerve, demonstrating that Translarna™ (ataluren) improved clinical outcomes of patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) in males five…

Endogenous Stem Cells Studied as Muscular Dystrophy Treatment

Insights into recent advances for Muscular dystrophy disease treatment, entitled “HDAC-regulated myomiRs control BAF60 variant exchange and direct the functional phenotype of fibro-adipogenic progenitors in dystrophic muscles” were recently published in the Genes & Development journal by Dr. Valentina Saccone. Dr. Saccone is part of Dr. Pier Lorenzo Puri’s…

Immune Cells May Help Treat Muscular Dystrophy, UCSF-Led Team Finds

An international research team led by University of California at San Francisco scientists has discovered that regulatory T cells (Tregs), a specialized subset of immune cells, suppress inflammation and muscle injury in a mouse model of Duchenne muscular dystrophy (DMD). The scientists say Tregs have potential as therapeutic agents…

New Guidelines Recommend Genetic Testing on MD Patients

The American Academy of Neurology (AAN) and the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) recently released new guidelines on the evaluation of MD patients by physicians, recommending that professionals should review all facts regarding symptoms, family history, ethnicity, physical exams, and lab tests in order to determine the…