News

Crowdsourcing Contest Seeks Algorithms for ALS Therapy Improvement

Investigators from the Helmholtz Zentrum München and Ludwig-Maximilians-Universität München in Germany are organizing a crowd sourcing exercise as a way of determining new algorithms that they expect will be able to accelerate their research into treatment for amyotrophic lateral sclerosis (ALS). The challenge itself, as well as the most effective algorithms found, were recently published…

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…