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Toward ‘Responsive’ Outcome Measures for DM1

Discovery of the holy grail for DM therapy development-- drug registration endpoints -- lies in the dogged pursuit and sharing of natural history data.

GSK3β as a Drug Development Target for DM1

A new study examines the role of the GSK3β—cyclin D3—CUGBP1 pathway in the pathogenesis of DM1 and its potential as a therapeutic target.

A Tool for Evaluating Drugs Targeting MBNL

Development of a mini gene tool facilitates the identification of candidate therapeutics targeted at dissociating MBNL from expanded CUG repeats.

NIH to Release RFA to Re-Compete Rare Disease Clinical Research Network

NIH announces intent to reissue a Funding Opportunity Announcement for the Rare Disease Clinical Research Network competition.

Skin Cancer Risks in DM1

Increased skin cancer risks in DM1 are captured in a new, longitudinal electronic medical records study.

Search for Prognostic Biomarkers for DM Cardiac Conduction Abnormalities

A recent publication suggests that a serum protein may ultimately have value as a biomarker for conduction system abnormalities in DM.

Finding the Right CRISPR Targets for DM

CRISPR is an exploratory strategy with potential for treatment of RNA-triggered diseases. Will DNA or RNA targeting prove to be the best approach for DM?

IL-6 Myokine Signaling in CDM

Genetic and epigenetic mechanisms drive differences between CDM and DM1—a new study elucidates downstream signaling pathways that underlie their diverse phenotypes and represent putative therapy development targets for CDM.

Interaction of Age and Gender in DM2

Age and gender impact the onset and progression of DM2, but the pattern shows both similarities and differences from that of DM1.

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.

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