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Telling the Quacks from the Cures

Many of you have seen posts on social media about treatments they have received in other countries or heard about through friends, that include everything from dietary aids to gene therapy, and want to how you can assess the possible benefits and risks of these 'treatments.' In this complicated therapy environment, how can patients make decisions about whether an available treatment or therapy is safe and effective? How can you tell the quacks from the cures?

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.

MDF is pleased to announce the following grant awards in partnership with the Wyck Foundation, London, UK.

MDF and Wyck jointly announce the funding of two new research projects. The projects address critical gaps in research infrastructure and clinical trial readiness and will increase understanding of the progression of DM, and provide measures to evaluate disease progression and the efficacy of candidate therapeutics.

 

International Expert Team Meets to Finalize First-Ever DM Care Recommendations for Doctors

MDF and its international team of clinical care partners just met in Miami to draft consensus-based Care Considerations for doctors, pharmaceutical companies and federal regulators reviewing potential therapies. Read more in this article.

New Findings on Quality of Life in DM2

A new quality of life study found that some DM2 patients are impacted as severely as those with DM1. Read more on the findings here.

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