Biotherapeutics company aTyr Pharma, Inc. was recently awarded orphan drug designation from the European Commission (EC) for its Resolaris therapy, designed to treat facioscapulohumeral muscular dystrophy (FSHD). The investigational drug is the company’s first Physiocrine-based therapy, and is currently being developed to become a first-in-class intravenous protein treatment for…
The Muscular Dystrophy Association (MDA) and Lowe’s stores are joining efforts to launch the MDA’s Shamrock program, the largest St. Patrick’s Day philanthropic event dedicated to help patients who live with the disabling muscle disease Duchenne muscular dystrophy. The FORTUNE 100 home improvement…
Dr. Thomas Gionis The use of stem cell therapy for a wide range of diseases continues to gain momentum across the United States, with more and more patients and their families seeking information about the benefits and potential risks of the treatment. In answer to this, clinics such as…
The Muscular Dystrophy Association (MDA) has released a report on the latest findings and developments on treatments for Duchenne muscular dystrophy that combat fibrosis and heart conditions associated with the disease. The Drug Development for DMD: February 2015…
A new study entitled, “Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers” was recently completed through a joint collaboration between researchers from the CNRS, UVSQ and INSERM at both the Laboratoire END-ICAP and the University of Bern. They discovered that through RNA “surgery,” synthetic oligonucleotides may…
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,…
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.
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