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What We Do (and Don’t) Know About Cognitive Function in DM1

A new literature review suggests meta-analysis of cognitive studies in DM1 is both feasible and can inform understanding of disease mechanisms and patient management.

The Origin of Insulin Resistance in DM

It would be easy to conclude that insulin resistance and diabetes in myotonic dystrophy is a product of mis-splicing of INSR transcripts—but is that the full story?

RAN Translation in DM2

Toxic RNA-mediated mis-splicing and Repeat-Associated Non-AUG (RAN) translation proteins are impacted by free MBNL1 levels and contribute towards CNS pathogenesis in DM2.

Interim Results Released for AMO Pharma Clinical Trial

AMO Pharma released interim results for a Phase 2a safety and toxicity study of Tideglusib (also known as AMO-02) for adolescents and adults with congenital myotonic dystrophy.

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.

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