A novel genome editing strategy removes expanded CUG repeats in DMPK transcripts.
DTI detects specific changes in the corticospinal tract, reflecting cortical gray matter volume reductions linked to DM1 motor function deficits.
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.