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01/07/2018 - 3:51pm

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

01/07/2018 - 3:43pm

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.

01/07/2018 - 3:29pm

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

12/11/2017 - 2:27pm

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

12/11/2017 - 2:06pm

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

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