Randolph

Facioscapulohumeral muscular dystrophy (FSHD) is a genetic condition that causes ongoing weakening of skeletal muscles. This research investigates a patient-driven health platform and registry called BetterLife FSHD, designed to support people living with FSHD by connecting them to personalized resources, tools, and relevant research opportunities. The platform also collects secure health and experience data to better understand the disease and improve care and treatments. Participants in BetterLife FSHD complete a series of short surveys at quarterly and yearly intervals. These surveys cover topics like demographics, health history, diagnosis and progression of FSHD, management strategies, and quality of life factors such as pain, fatigue, and mental health. Based on their responses, participants receive personalized resources and are informed about clinical trials and research studies they may qualify for. The collected data is securely stored and shared in a de-identified form with approved researchers and organizations. Throughout the study, participants provide health information regularly through surveys over a period of up to 10 years. The research team assesses longitudinal health data annually and tracks self-reported FSHD progression every six months. Additional patient-reported outcomes related to anxiety, depression, pain, sleep, fatigue, mobility, physical activity, falls, and other health aspects are collected quarterly or yearly. This ongoing data collection supports a comprehensive understanding of living with FSHD and advances research efforts.