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Developing Therapies for Brain-related DM Symptoms - Add Your Voice

MDF and IDMC will co-host a moderated multi-panel presentation entitled “Bringing the Patient Voice to CNS-Targeting Drug Development in Myotonic Dystrophy,” at the 2017 MDF Annual Conference on Saturday, September 8th. MDF and IDMC developed the presentation in response to requests from drug development professionals to help identify CNS (brain-related) DM symptoms and what CNS-targeting DM therapies should do, as defined by people living with DM

The meeting format will mirror the very successful Patient-Focused Drug Development (PFDD) meeting hosted by MDF with the FDA in September 2016. Panels will be seated with people affected by DM and caregivers. The session will be moderated by James Valentine, J.D., who also moderated the September 2016 PFDD meeting. 

The session will begin with a presentation on outcome measure development to help the lay audience understand the importance and role of endpoints in drug development and approval, and frame the goals for the session. Panel presentations will then be followed by moderated audience discussion and a presentation from an industry representative regarding possible future DM CNS endpoints, and CNS endpoints used in other neurological diseases that might inform upcoming efforts in DM.

The 2017 MDF Annual Conference will gather more than 650 families, clinicians, researchers, industry representatives and caregivers working to improve the lives of people living with myotonic dystrophy. Learn more about 2017 MDF Annual Conference sessions, and register.

 

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