Home / News / Research

Research

Risk of GI Manifestations in DM1 and DM2

New data is available on the frequency, progression and treatment of GI symptoms in DM1 and DM2.

Current Status of Brain Imaging in DM1

A new review article assesses the current status of and recommends future directions for brain imaging studies in DM1.

Understanding DM1 Patient Perceptions about Their Disease

Given the brain manifestations of DM1, how aware are most patients of the impact and progression of their disease?

Made to Measure: Developing Clinical Tools to Capture the Severity and Progress of DM

Learn more about Dr. Ami Mankodi, principal investigator at the National Institutes of Health’s (NIH) National Institute of Neurological Disorders and Stroke (NINDS) in Bethesda, Maryland. Dr. Mankodi has been involved in research that has helped shape a fundamental biologic and molecular understanding of myotonic dystrophy (DM).

Modifying Gene Editing Technology for DM

Investigators at the University of California San Diego, the University of Florida, and the National University of Singapore have recently reported early research that potentially ‘repurposes’ gene editing technology for a set of RNA disorders—myotonic dystrophy type 1 (DM1), myotonic dystrophy type 2 (DM2), a subset of Lou Gehrig’s disease (ALS) patients and Huntington’s disease. They have modified the Cas9 enzyme so it is targeted to toxic RNA, instead of the expanded DNA repeats in these diseases.

Gene Editing Repurposed Toward Toxic RNA

A novel redirection of CRISPR/Cas9 technology addresses toxic RNA, rather than the genome, as a potential therapy for DM1 and DM2.

Know Your DM1 Repeat Length: It’s Important for Your Cardiac Care

Understanding cardiac and other DM risk factors and planning for the known complications of DM that may affect you can help protect and maintain your quality of life and that of your loved ones.

Molecular Events Underlying Congenital DM

A new study points to the timing of MBNL-dependent RNA processing defects as a major factor in the pathogenesis of CDM.

Understanding and Measuring Fatigue in DM1

Although fatigue represents a substantial burden in DM1, tools must be validated to assess its diverse contributing factors in order to develop clinical trial endpoints and effective therapies.

MDF at 2017 BIO International Convention

MDF staff recently attended the 2017 BIO International Convention hosted by the Biotechnology Innovation Organization (BIO). BIO offers a partnering track to patient advocacy groups, enabling meetings with companies having potentially mutual interests.

Partners

© Myotonic Dystrophy Foundation. All rights reserved.