A new study points to the timing of MBNL-dependent RNA processing defects as a major factor in the pathogenesis of CDM.
Although fatigue represents a substantial burden in DM1, tools must be validated to assess its diverse contributing factors in order to develop clinical trial endpoints and effective therapies.
In June of this year, MDF initiated a new program called “MDF DM Days” to bring together clinicians, researchers and myotonic dystrophy (DM) family members, and provide close-to-home access to educational sessions.
MDF and the International Myotonic Dystrophy Consortium (IDMC) Will Co-host a Workshop on CNS Endpoints for DM Clinical Trials - Add Your Voice
At the 2017 MDF Annual Conference, the Myotonic Dystrophy Foundation (MDF) will offer MDF community members the opportunity to learn first-hand about U.S. federal and state benefits that can be used to support individuals affected by myotonic dystrophy.