Work plans, strategic assessment and funding are all part of the annual strategic planning offsite MDF conducts every January. Learn more here.
Lukasz Sznajder, Ph.D., is developing a mouse model for type 2 myotonic dystrophy, a crucial step that is expected to advance understanding of and therapy for this disease.
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.