News

BioBlast Pharma Ltd. presented the preliminary results from its HOPEMD Phase 2/3 study investigating Cabaletta (trehalose) in patients with Occulopharyngeal Muscular Dystrophy (OPMD), a progressive, incapacitating genetic disease. Cabaletta is a chemical chaperone that has been shown to reduce pathological aggregation of proteins within cells in several diseases…

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…

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…