Clinical Trials for Myotonic Dystrophy Have Begun

ISIS Pharmaceuticals is developing a drug (ISIS-DMPKRx) to treat Myontonic Dystrophy Type 1 (DM1, also called Steinert’s Diseases). DM1 is a dominantly inherited, degenerative disorder that affects many systems in the body. DM1 is mainly characterized by progressive muscle wasting, weakness, and myotonia, but additional clinical features include early cataracts, cardiac conduction dysfunctions, hypersomnia, gastrointestinal abnormalities, insulin insensitivity, and infertility. DM1 is estimated to affect 1 in 8,000 people worldwide, or approximately 150,000 patients in the US, Europe and Japan. Currently, there are no disease-modifying therapies for patients with DM1 and treatments are intended only to manage symptoms. ISIS-DMPKRx is an antisense drug being developed for the potential treatment of DM1. ISIS-DMPKRx is currently being studied in a Phase 1 study in healthy volunteers to evaluate its safety. 

Learn more: print the trial fact sheet