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.
The Muscular Dystrophy Association’s 18th Annual Muscle Team Gala & Benefit Auction will take place this Tuesday, January 6 and will include athletes from Metro-New York area sports teams such as the New York Giants, New York Jets, and New York Red Bulls, plus representatives from the New York Aqueduct Racetrack, Muscle Team…
Researchers at the Ohio State University Wexner Medical Center, along with experts at several other medical facilities, have found that Duchenne muscular dystrophy (DMD)-related declines in heart functioning may be slowed by already existing heart medications. The research appeared in the online version of The Lancet Neurology. DMD is characterized by…
Patients suffering from muscular dystrophy may soon be able to independently monitor their therapeutic response to medications with a revolutionary smartphone-linked device. The Acoustical Society of America invited the creators and researchers behind this device to their annual meeting last October 30, 2014 to give a…
South Plainfield, NJ-based biopharmaceutical company PTC Therapeutics, Inc. recently announced that it has launched a rolling submission of a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for pipeline nonsense mutation Duchenne muscular dystrophy (nmDMD) treatment Translarna™. This process enables…
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