Researchers from Dr. Matthew Disney's lab at the Scripps Research Institute of Florida recently published an article describing a new chemical they designed to inhibit the unhealthy repeat-containing RNA molecule seen in DM2.
Ionis Pharmaceuticals, Inc. (formerly Isis Pharmaceuticals, Inc.) announced today that it has launched a Phase 1 clinical trial for IONIS-DMPKRX. IONIS-DMPKRX is designed to reduce the production of toxic dystrophia myotonic-protein kinase (DMPK) RNA in cells, including muscle cells, for the treatment of Myotonic Dystrophy Type 1 (DM1).
Stanford University has launched the DM Biobank to provide high-quality DM biological samples to researchers around the world. By participating in the Biobank, DM community members can play an important role in the development of new therapies that may help patients today and for generations to come.
A recently published study from Sweden reported impaired facial recognition in people with DM1 and indicated that there are brain differences that affect how faces are perceived and stored by people with DM1.