Review
Aug 2026
Myotonic dystrophy type 1 - updates on mechanisms of multisystemic manifestations, genetically targeted therapeutics, and preparing for a postapproval world.
This review highlights genetically targeted therapeutics showing impact for muscle manifestations of DM1, ongoing efforts now shifting to expand to other target tissues and the younger population, and the understanding of societal burden and population genetics of DM1.
Samuel Carrell, Adam D. Comer, Nicholas E. Johnson
· Current Opinion in Neurology · 1 citation