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.
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 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.