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DM1 Genotype and Cardiac Phenotype

A large retrospective study helps clarify genotype-cardiac phenotype relationships in adult DM1.

Do We Treat DM as a Brain Disease?

A recent review article makes the case that DM is a brain disease and that better understanding of and treatment strategies for the neurological consequences of DM are essential.

The Path We’re On: Many Challenges, but Ultimately a Drug

Taking as many shots on goal as possible is the only path to approval and reimbursement of safe and effective drugs for DM.

Telling the Quacks from the Cures

MDF is also happy to help you understand whether something is in a legitimate clinical trial, an approved therapy…or not.

Myotonic Dystrophy Foundation Statement Regarding: American Health Care Act

In March, Republican Members of the U.S. House of Representatives’ Committee on Ways and Means and the U.S. House Committee on Energy and Commerce introduced legislation, entitled the American Health Care Act (AHCA), that would repeal and/or replace major parts of the Affordable Care Act (ACA).

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.

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.

Kidney Dysfunction is a Risk in DM1: How to Best Assess It

Since DM1-related loss of muscle mass confounds a common biomarker of kidney dysfunction, creatinine levels, serum cystatin C provides a superior measure of renal dysfunction for both patient management and clinical trials.

 

Gender-Related Cancer Risk in DM1

A recent study corroborated increased susceptibility to cancer in DM1, for women in particular, and linked the elevated risk to depressed levels of a tumor suppressor microRNA (miRNA). 

Singer Raises DM Awareness with Album Launch

Acclaimed musician Eric Hutchinson is using his new album to support Care and a Cure. Read his inspiring family story.

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