Ionis Pharmaceuticals recently announced completion of the IONIS-DMPKRx Phase 1/2a clinical trial, and stated that it expects to have analysis of the data collected through that study completed by early January 2017. The study was designed to evaluate the safety and tolerability of multiple doses of an investigational compound, IONIS-DMPKRx, in adult patients with DM1.
Throughout the year MDF has dispatched members of the team to local events around the country to raise disease visibility and distribute resources. Here is a recap of the different events we have attended so far this year.
MDF is celebrating a milestone anniversary: Ten years of driving Care and a Cure for myotonic dystrophy via programs that now serve more than 10,000 patients and family members per year in 62 countries.
In a recent study by Dr. Richard Moxley, III, MD, and colleagues at the University of Rochester, researchers assessed disease manifestations and adherence to medications for DM1 and DM2 patients. The study was motivated in part by the fact that DM patients need to take multiple prescriptions to manage disease symptoms associated with a number of different body systems.
Laura Renna, Ph.D., thinks there may be more to the insulin resistance story in myotonic dystrophy than abnormal insulin receptor splicing. She intends to probe the question by testing three insulin mimetics in cells taken from DM patients and age-matched controls.