News

Marathon Pharmaceuticals, LLC, a biopharmaceutical company currently developing treatments for rare diseases, recently announced that the FDA granted the Fast Track designation for their deflazacort drug to treat Duchenne Muscular Dystrophy (DMD). In addition, the company is also developing several strategies to address neurological movement and muscular disorders. DMD is a fatal…

Non-profit organization Parent Project Muscular Dystrophy (PPMD) has nominated John Porter, PhD. as its new CEO. The former program director of the National Institute of Neurological Disorders and Stroke at National Institutes of Health (NIH) will join the organization to assist in supporting research projects working towards…

Last week in Europe, Sarepta Therapeutics announced the initiation of their first human trial for the new drug SRP-4053 for Duchenne Muscular Dystrophy (DMD). SRP-4053 has shown remarkable promise in treating this debilitating childhood disease in the laboratory, and researchers are highly optimistic of the potential outcomes of a human trial.

Based on the fact that in vitro models of human skeletal muscle cannot recapitulate human muscle functions, a team of researchers from Duke University developed human muscle tissues called “myobundles” using primary myogenic cells. The researchers observed that these muscle tissues were able to respond to electrical stimuli…

Sarepta Therapeutics, Inc, a company focused on developing RNA-based therapeutics, recently announced their findings through Week 168 from Study 202, a phase IIb study that is evaluating the efficacy of a drug called eteplirsen in patients with Duchenne muscular dystrophy (DMD). The company’s study 201 was a randomized, double-blind,…

Last year, 2014, an inspiring 9-year-old girl from Wisconsin travelled all over the United States as one of the ambassadors of the Muscular Dystrophy Association. This year, she resumes her advocacy as a National Goodwill Ambassador, determined to strengthen awareness and support for muscular dystrophy, which…

Tivorsan Pharmaceuticals, Inc. (Tivorsan), a protein therapeutics company with a focus on developing treatments for neuromuscular disorders such as muscular dystrophy, recently announced the company has achieved a fourth set of pivotal milestones under a series of research grants from Parent Project Muscular Dystrophy (PPMD) and Muscular…

PTC Therapeutics, Inc. submitted a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for Translarna™. The medication is used for treating nonsense mutation Duchenne muscular dystrophy (nmDMD). Duchenne muscular dystrophy (DMD) is a progressive disorder of the muscles that mostly affects males. It is caused by a…

After the weeding, the hospital offered the couple a private room. The love story between 37-year old Chris Plum and 42-year old Margaret or “Muffi” Lavigne is already special, as they met and were married two years later at the Hospital for Special Care in New Britain, Connecticut, where they…

A study led by a cardiologist at The Ohio State University Wexner Medical Center found that administering medication for heart failure as early as possible can help slow progressive decline in cardiac function, commonly observed in young boys diagnosed with Duchenne muscular dystrophy (DMD). Lead author Dr.