Patient-Focused Drug Development Meeting, Part 2 (2016 Myotonic Annual Conference)

Myotonic community members living with DM and their caregivers present on current symptom management strategies for living with DM, and what they would like to see in clinically-meaningful therapies. Dr. Jonathan Goldsmith, M.D., FACP, head of the Office of Rare Diseases at the FDA, provided a summary of the proceedings and what the FDA took away from the patient input shared at this meeting – at the 2016 DM Patient-Focused Drug Development meeting, a component of the 2016 Myotonic Annual Conference.