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.
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.
