The Myotonic Dystrophy Foundation is currently planning a workshop to address CNS involvement in DM1, DM2 and CDM, to be held on September 12, 2019, in conjunction with the 2019 MDF Annual Conference, September 13-14, in Philadelphia, PA.
The Workshop will bring together stakeholders in academia, the biotechnology/pharmaceutical industry, and regulatory agencies for a day-long discussion of opportunities and hurdles to overcome in therapy development efforts for the CNS phenotype in DM.
Specifically, the Workshop will examine the natural history of DM1, DM2 and CDM and the underlying scientific premise for targeting therapy development to address the CNS phenotype, a key contributor to overall burden of disease. Through platform presentations and focused discussion sessions, the Workshop will seek to identify targetable CNS phenotypes, evaluate the current knowledge of the underlying disease mechanisms and molecular targets, and begin to determine how interventional trials can best demonstrate the clinical effectiveness of drugs and biologics in patients living with DM1 and CDM. Taken together, the Workshop will identify opportunities and knowledge gaps to guide improvements in clinical trial readiness for mitigating the critically important CNS sequalae of the disease.