Development, validation, and use of CNS clinical outcome assessments in DM1 must consider their interactions with other parameters of patient functioning.
An innate immune response pathway, the induction of interferon type 1, rather than activation of autophagy, is responsible for impaired myogenesis in CDM.
The Myotonic Dystrophy Foundation has hired a branding and design agency, A Line Studio, to review the MDF brand (our logo, name and the visual identity that defines us) and determine how to best remove the confusion between MDF and other organizations, and evolve and breathe new life into the MDF brand.
A literature review suggests meta-analysis of cognitive studies in DM1 is both feasible and can inform understanding of disease mechanisms and patient management.
A new study lends support for a DM biomarker panel based upon patient urine exRNA assays that may serve as a pharmacodynamic biomarker for go/no go decisions in drug development for DM1.