News

Cambridge, Massachusetts based clinical stage biopharmaceutical company Acceleron Pharma Inc. has announced its intent to offer and sell, subject to market and other conditions, $150 million of the company’s common stock in an underwritten public offering. Acceleron intends to use net proceeds from the offering partly to conduct clinical trials…

Researchers from Duke University have succeeded in treating an adult mouse model of Duchenne muscular dystrophy using the gene editing system CRISPR — the first successful treatment of a genetic disease in an adult animal utilizing a method that has the potential to be used in humans. In spite of recent advances…

Impaired cellular energy metabolism has been widely reported as a key element in dystrophin-deficient muscle degeneration, one of the most significant aspects of Duchenne Muscular Dystrophy (DMD). In a review entitled “Metabogenic and Nutriceutical Approaches to Address Energy Dysregulation and Skeletal Muscle Wasting in Duchenne Muscular Dystrophy,” the authors present…

UT Southwestern has been awarded a $7.8 million, five-year grant to establish the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, with a special focus on inherited Duchenne muscular dystrophy (DMD). “UT Southwestern is the perfect environment for the Wellstone Center. We can merge cutting-edge science with clinical application,” said Dr.

Throughout 2015, Muscular Dystrophy News covered the latest developments in science, research, and advocacy news in muscular dystrophy. As the year comes to an end, here are the 10 articles most widely read by Muscular Dystrophy News readers, each with a brief summary of the developments that made them of such…

Researchers have identified two promising biomarkers for the noninvasive monitoring of Duchenne muscular dystrophy (DMD) disease progression or regression, although more research is necessary. The paper, titled “Comparative mass spectrometric and immunoassay-based proteome analysis in serum of Duchenne muscular dystrophy patients,” was published in Proteomics-Clinical Applications. Duchenne muscular dystrophy…

SOM Biotech announced the start of its first crowdfunding campaign to initiate new R&D projects into rare pediatric diseases, including Duchenne muscular dystrophy (DMD). The company, headquartered at the Barcelona Science Park (PCB), aims to raise at least $300,000 to be divided evenly among five rare childhood diseases: DMD, cystic fibrosis, Gaucher’s disease (…

Solid GT, LLC, announced that it has received has $42.5 million in Series B venture capital financing, which will be used to further the develop the company’s novel gene therapy platform for Duchenne muscular dystrophy (DMD). “We are proud of this pivotal milestone for Solid GT and our partnership with such leaders of industry,”…

CITGO Petroleum Corporation announced it has partnered with the Muscular Dystrophy Association (MDA) to support its MDA Arkansas Chapter “Passport to a Cure” event. The Hollywood-themed event collected more $30,000 in funds, which will be used to benefit research and local families dealing with the neuromuscular disease. The MDA currently serves over 800 people in…

Researchers have shown for the first time that a cancer-treating drug is able to neutralize the toxic RNA responsible for prolonged muscle contractions and other symptoms in myotonic dystrophy type 1. The study, titled “Actinomycin D Specifically Reduces Expanded CUG Repeat RNA in Myotonic Dystrophy Models,” was published…