A novel genome editing strategy removes expanded CUG repeats in DMPK transcripts.
TREAT-NMD’s TACT process provides unbiased feedback on drug development programs in neuromuscular diseases.
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.
An innate immune response pathway, the induction of interferon type 1, rather than activation of autophagy, is responsible for impaired myogenesis in CDM.