A large natural history study of children with DM1 may form the background for subsequent development of evidence-based case management guidance.
A new review article synthesizes published data on health-related quality of life in adult-onset DM1.
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.