DM patient and family member/caretaker assessments of the burden of disease form the basis of a recent publication.
A new longitudinal study focuses on CNS function in congenital and childhood DM1.
A new review article addresses advances in exploiting the biology of RNA with small molecule drug strategies.
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.