MDF will host a Workshop on CNS involvement in DM and the development of outcome measures for CNS-targeting therapies on September 12, 2019, in conjunction with the MDF Annual Conference, September 13-14, in Philadelphia, PA.
This 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 discuss what biomarkers and outcome measures might best demonstrate the clinical effectiveness of drugs and biologics in patients living with DM1, DM2 and CDM.
The meeting will take place from 8:00 AM to 4:00 PM. Lunch will be provided.