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.
Stanford University has created the Myotonic Dystrophy Biobank to aid researchers in advancing therapies.
In early May, the Foundation announced a Request for Applications for this year's MDF Fund-a-Fellow program. The Fund-a-Fellow program supports our commitment to Care and a Cure for myotonic dystrophy by attracting new investigators to the field of DM research, ultimately advancing DM science and the search for treatments.
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.