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.