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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.

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.

Treating Sleep Disorders in DM1

Sleep studies are indicated for DM1 patients with disrupted sleep or daytime sleepiness in order to direct selection of the optimal treatment regimen.

AMPK/mTORC1 Signaling as a Therapeutic Target for DM1

Studies of AMPK/mTORC1 signaling in DM1 identify novel therapeutic targets for DM, and may offer an opportunity to repurpose approved drugs for both muscle and cognitive symptoms.

MDF Finished Year 10 with Flying Colors

2016 was a banner year for the Myotonic Dystrophy Foundation. We completed our 10th year driving Care and a Cure for people and families living with DM, wrapped up year 2 of a 3-year, $5M drug development acceleration effort and oversaw the largest expansion of our Care programming and resources ever. 

Toward Effective Biomarkers for DM

Biomarkers of various Contexts of Use are essential for drug development in DM—recent guidance documents and publications point to exciting new opportunities.

Cognitive Changes in Adult DM1: An Acceleration of Normal Aging?

Longitudinal assessment of cognitive function in adult- and late-onset DM1 reveals a pattern of cognitive decline that can be modeled as an early-onset and acceleration of normal aging.

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