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.
New research on the impact of the mother's age on congenitally affected offspring.
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.
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).