Small molecules which improve pathogenesis of myotonic dystrophy type 1
(2018)
Journal Article
López-Morató, M., Brook, J. D., & Wojciechowska, M. (2018). Small molecules which improve pathogenesis of myotonic dystrophy type 1. Frontiers in Neurology, 9, Article 349. https://doi.org/10.3389/fneur.2018.00349
Myotonic dystrophy type 1 (DM1) is the most common muscular dystrophy in adults for which there is currently no treatment. The pathogenesis of this autosomal dominant disorder is associated with the expansion of CTG repeats in the 3′-UTR of the DMPK... Read More about Small molecules which improve pathogenesis of myotonic dystrophy type 1.