Objective Monitoring of Facioscapulohumeral Dystrophy During Clinical Trials Using a Smartphone App and Wearables: Observational Study.

Maleki G, Zhuparris A, Koopmans I, Doll RJ, Voet N, Cohen A, van Brummelen E, Groeneveld GJ, De Maeyer J

Facioscapulohumeral dystrophy (FSHD) is a progressive muscle dystrophy disorder leading to significant disability. Currently, FSHD symptom severity is assessed by clinical assessments such as the FSHD clinical score and the Timed Up-and-Go test. These assessments are limited in their ability to capture changes continuously and the full impact of the disease on patients' quality of life. Real-world data related to physical activity, sleep, and social behavior could potentially provide additional insight into the impact of the disease and might be useful in assessing treatment effects on aspects that are important contributors to the functioning and well-being of patients with FSHD.