A new study entitled “Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers” shows a new class of antisense oligonucleotides – tcDNA- antisense oligonucleotides – are highly efficient at rescuing dystrophin levels and improving symptoms in a mouse model of Duchenne muscular dystrophy.
The Muscular Dystrophy Association recently announced the start of the 13th annual “Appetite for a Cure” campaign to raise critically-needed funds and mobilize awareness to help the MDA improve the lives of both children and adults who live with muscular dystrophy. From February 2 through March 22, more than 130 Sailormen/Popeye’s restaurants will participate…
Nominations for individuals who wish to serve as non-federal public members on the Muscular Dystrophy Coordinating Committee (MDCC) are currently open and being accepted by the office of the Secretary of the Department of Health and Human Services (HHS) until February 27. The MDCC is a federal advisory commission that was created…
The nonprofit organization, Parent Project Muscular Dystrophy (PPMD), has announced it will award a $50,000 exploratory grant to a research team from UCLA‘s David Geffen School of Medicine and College of Letters and Science, led by Dr. M. Carrie Miceli. The funding will support the study of exon skipping boosters for the treatment of DMD,…
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…
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