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Rare Chemistry: Matt Disney Advances Development of Small Molecule Therapeutics for DM

Dr. Matt Disney brings an unusual and increasingly valuable skill to therapy development for DM—he’s a chemist. The NIH has recently awarded two research grants in support of Dr. Disney’s research. Read this article to find out how his work leverages the latest advances in RNA biology to target the unique disease mechanisms of DM.

Gender Matters in DM1

Dr. Guillaume Bassez and a large team in France and Canada recently published an analysis of gender and its impact on the DM1 phenotype. Read about their findings in this article.

Ionis Pharmaceuticals Reports on Phase I & I/II Ionis-DMPKrx Studies

Dr. Laurence Mignon, Director of Clinical Development at Ionis Pharmaceuticals in Carlsbad, CA, and Dr. Charles Thornton, neurologist and a principal investigator at the University of Rochester, reported on the status of the Phase I and Phase I/II clinical trials for IONIS-DMPKRx, a potential DM therapy, at the annual meeting of the American Academy of Neurology in Vancouver, BC last month.

Molecular Changes Lead to Cardiac Dysfunction in DM

A new study by a multi-disciplinary, international team has shed light on the molecular changes at the root of DM-related heart dysfunction, the second leading cause of death for DM patients.

The Genetics of DM1 Repeat Size

Researchers from the University of Costa Rica and the University of Glasgow teamed up to investigate the DNA mutation causing myotonic dystrophy type 1 (DM1). They found that polymorphism in the MSH3 mismatch repair gene is associated with the levels of somatic instability of the expanded CTG repeat in the blood DNA of myotonic dystrophy type 1 patients. 

MDF Research Fellow Profile: Dr. Melissa Dixon

Dr. Dixon at the University of Utah has been awarded a 2016-2017 postdoctoral fellowship for her research applying fMRI to evaluate connectivity networks in the brains of children with congenital-onset myotonic dystrophy (CDM). Read more here about the work of Dr. Dixon and her colleagues.

MDF Welcomes Dr. John Porter as Interim Chief Science Officer

Dr. John Porter, former Program Director of Neuromuscular Disease at the National Institute of Neurological Disorders and Stroke (NINDS), a component of the National Institutes of Health (NIH), has joined MDF as its Interim Chief Science Officer. Dr. Sharon Hesterlee, who formerly filled this position, will remain active as a member of MDF’s Scientific Advisory Committee.

Dr. Charles Thornton Wins Prestigious Javits Award

Dr. Charles Thornton, neurologist at the University of Rochester Medical Center and MDF Scientific Advisory Committee member, has been awarded a Javits Neuroscience Investigator Award from the National Institutes of Health to further his research on muscular dystrophy. Read more about this very distinguished award for “exceptional” investigators and the important work Dr. Thornton is doing here.

University of Iowa Launches Brain Imaging Study

The University of Iowa has been awarded a grant by the National Institute of Neurological Disorders and Stroke (NINDS) to fund a 3-year longitudinal study of adults with a family history of DM1. Learn more about the study and how to participate in this article.

 

Industry Corner: Cloud Pharmaceuticals

Cloud Pharmaceuticals, a cloud-based drug design and development company, is interested in potential collaborations around myotonic dystrophy targets.

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