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.
NINDS announces its intent to issue Funding Opportunity Announcements to support analytical validation of identified candidate biomarkers.