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MDF Launches First MDF DM Day in Iowa

MDF is pleased to announce the launch of a new program, MDF DM Days. These special one-day free events will be held in three locations across the U.S. in 2017, bringing together clinicians, researchers and MDF community members to share information and resources.

Epigenetics Underlying the Parent of Origin Effect in CDM

Epigenetic modifications upstream of an expanded DMPK allele may underlie the maternal bias in the inheritance of CDM.

A Biomarker for Cardiac Dysfunction in DM1?

A multi-center study suggests serum cardiac troponin-1 levels predict risks of left ventricular dysfunction in DM1 patients.

Using Gene Editing to Correct DM

A potentially revolutionary technology may allow development of a drug for DM that can correct a patient’s DNA by selectively removing the expanded CTG and CCTG repeats in DM1 and DM2, respectively.

Adequate Rationale to Launch Clinical Trials

Clinical trials involve substantial investments by drug developers, but especially by DM patients and families. Clinical trials proposed for our community must have adequate scientific rationale behind them.

Changes in NINDS Postdoctoral Training Initiatives

MDF runs down some important changes in the purposes and rationale of NINDS funding mechanisms for postdoctoral fellows.

FDA: How to Talk with Us About INDs

FDA released an online course to accompany their draft guidance on how to best communicate with the Agency in preparing and filing an IND.

Gene Editing for DM

Gene editing is a potential avenue for therapy development in DM. With the safety, efficacy and delivery challenges, how do we get there?

Improving Clinical Trials in Myotonic Dystrophy: Thurman Wheeler, M.D.

When Dr. Thurman Wheeler was a resident in neurology, he remembers a senior physician telling him that myotonic dystrophy would probably be one of the most difficult diseases to treat because it involves so many body systems.

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