Discovery of the holy grail for DM therapy development-- drug registration endpoints -- lies in the dogged pursuit and sharing of natural history data.
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.
Development of a mini gene tool facilitates the identification of candidate therapeutics targeted at dissociating MBNL from expanded CUG repeats.
NIH announces intent to reissue a Funding Opportunity Announcement for the Rare Disease Clinical Research Network competition.
Increased skin cancer risks in DM1 are captured in a new, longitudinal electronic medical records study.
A recent publication suggests that a serum protein may ultimately have value as a biomarker for conduction system abnormalities in 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?
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.
Age and gender impact the onset and progression of DM2, but the pattern shows both similarities and differences from that of 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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