A new study lends support for a DM biomarker panel based upon patient urine exRNA assays that may serve as a pharmacodynamic biomarker for go/no go decisions in drug development for DM1.
A new review looks at the cross-disease evidence for RAN translation’s contributions to pathogenesis.
New evidence links mis-splicing of Scn5a to cardiac conduction defects and arrhythmias in DM.
When Haukur Svansson began studying neurological disease as part of his work towards an undergraduate degree in psychology at the University of Iceland in Reykjavik, he began to feel ashamed of himself for not knowing more about Vöðva og Spennuvisnun, the Icelandic term for myotonic dystrophy.
MDF and our community advocates have again successfully partnered with congressional leaders to include myotonic dystrophy in the Peer-reviewed Medical Research Program (PRMRP) of the U.S. Department of Defense.