If you have this disease call you local research center and ask to be added to list of potential patients if you want to participate.
Isis Pharmaceuticals Initiates Phase 1/2 Study of ISIS-DMPK Rx in Patients With Myotonic Dystrophy Type 1
ISIS-DMPK Rx Extends the Targeting of Antisense Drugs to Include Muscle Tissue
CARLSBAD, Calif., Dec. 16, 2014 /PRNewswire/ — Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has initiated a study for ISIS-DMPKRx in patients with Myotonic Dystrophy Type 1 (DM1). DM1 is a rare genetic neuromuscular disease caused by the production of toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells. ISIS-DMPKRx is specifically designed to reduce toxic DMPK RNA.