Rare disease patients and caregivers have played a critical role in helping to elevate the importance of focusing on the whole patient when treating a rare disease. Treatments and clinical care must include efforts to address the physical symptoms of rare disease patient communities and researchers are increasingly realizing that addressing just physical symptoms of rare diseases is insufficient. Clinical care must also focus on improving quality-of-life for patients and caregivers — physically, emotionally and socially. But how do we measure these impacts? Join this session to learn more about cutting-edge research to improve how we measure quality-of-life and how we can include these measures in research on therapy development and clinical care practices.
Speaker: Maria Picone, Founder and Chief Executive Officer, TREND Community, and Prader-Willi Syndrome Parent Advocate
Speaker: Tiffany Westrich-Robertson, Chief Executive Officer, International Foundation for Autoimmune & Autoinflammatory Arthritis
Day 1, Track 4