Development, validation, and use of CNS clinical outcome assessments in DM1 must consider their interactions with other parameters of patient functioning.
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.
A new review looks at the cross-disease evidence for RAN translation’s contributions to pathogenesis.
MDF is pleased to announce the publication of the first-ever Consensus-based Care Recommendations for Adults with Myotonic Dystrophy Type 1.