Researchers at the University of Illinois recently re-engineered small molecules to disrupt toxic RNA repeats involved in DM2.
The DMCRN was established a little over three years ago to evaluate potential DM treatments, prepare possible clinical trial sites and conduct important DM research studies.
Dr. Darren Monckton is an internationally-known DM researcher who provides valuable information to the MDF community. Check out his videos and upcoming webinar that will explore the significance of CTG repeats for people with DM and how to understand blood test results.
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).
Researchers identify the gene believed to be responsible for adverse statin drug side effects in DM2 patients.