Renowned lawyer and health advocate Kristin Stephenson was recently named by the Muscular Dystrophy Association (MDA) as the organization’s Vice President of Policy and Advocacy. Stephenson was already an active member of the MDA community and now, with her new responsibilities, she will be implementing, developing and overseeing government and advocacy relations strategies to better achieve…
aTyr Pharma, a biotherapeutics company committed to discovering and developing therapeutics for patients with severe rare diseases based on Physioncrine biology, recently announced the enrollment of the first patient with Facioscapulohumeral muscular dystrophy in their clinical trial of new drug called Resolaris™. Resolaris, is a first-in-class intravenous protein used for…
MRIGlobal, an independent, not-for-profit, contract research organization based in Kansas City, Missouri, announced earlier in January that it has received a 10-year research grant worth $54 million, care of the National Institute of Neurological Disorders and Stroke. The new funds will be allotted for the advancement and manufacturing of new therapeutics for…
A team of researchers from the University of Portsmouth recently found that in Duchenne muscular dystrophy, a phenomena called autophagy occurs where the damaged muscle cells destroy themselves. Dr. Chris Young discovered this mechanism. The research group led by Professor Darek Gorecki is working on a study focused on Duchenne muscular dystrophy.
Researchers at Nationwide Children’s Hospital in Columbus, Ohio have developed a method for measurement of upper extremity movement in muscular dystrophy patients that employs interactive video game technology. The objective is an expansion of inclusion criteria for patients using wheelchairs who participate in clinical trials. Duchenne muscular dystrophy,…
California-based biotech company, Capricor Therapeutics, Inc., recently held a webinar on Wednesday, January 21, 2015 on the company’s latest research and development updates on addressing the largely unmet clinical needs of Duchenne muscular dystrophy (DMD) and related heart disease, which is known to be the most common cause of mortality…
The Kids Wish Network, an international charitable organization that grants wishes to children with life-threatening medical conditions, recently congratulated one of its celebrity Guardian Angel Wish Sponsors for being selected to win a humanitarian award. American country singer and songwriter, Martina McBride, will receive the distinguished Harry Chapin Memorial Humanitarian Award from the Music…
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.
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