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News

01/09/2018 - 6:30am

MDF has been awarded a $1M donation by an anonymous family in the DM community to launch development of gene editing technology to pursue the search for a cure for DM1.

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.

01/07/2018 - 3:20pm

NIH has reissued the CREATE Bio, Blueprint Neurotherapeutics and NeuroNEXT initiatives, along with other initiatives that focus on biomarker development and clinical trial readiness, to support therapy development efforts.

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