CNS and Myotonic Dystrophy: Outcome Measures Development Workshop
Event Information
Description
Myotonic 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 Myotonic 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.
Click here to see the final agenda.
Questions?
Contact MDF at 415-800-7777 or via email.
Organizer Myotonic Dystrophy Foundation
Organizer of CNS and Myotonic Dystrophy: Outcome Measures Development Workshop
Our mission, "Care and a Cure," is to enhance the quality of life of people living with myotonic dystrophy (DM) and accelerate research focused on finding treatments and a cure.
The Myotonic Dystrophy Foundation (MDF) is the leading global advocate helping patients and families navigate the DM disease process, and is often the first resource contacted by newly-diagnosed patients, their families, their social workers and their physicians around the world. For many international patients, the Myotonic Dystrophy Foundation is often the only resource they are able to locate, and MDF has provided assistance and support to families in more than 80 countries as far-flung as Croatia, Jordan, Chile, New Zealand and Israel. Currently tens of thousands of people living with myotonic dystrophy, their families & friends make up the Myotonic Dystrophy Foundation community.
Learn more about MDF and myotonic dystrophy at www.myotonic.org.
