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 to capture the full impact of the disease on patients’ quality of life.
In our exciting new publication we demonstrated that real-world data, captured using a patient's own smartphone in combination with a simple wearable device, can provide important insight into the impact of disease of patients with FSHD. In another, soon to be published manuscript, we demonstrated that an algorithm based on the variables yielded by the smartphone and wearable device correlates well with disease severity. This tool, developed and validated with co-funding of CHDR's R&D budget, may turn out to be useful in assessing treatment effects on aspects that are important contributors to the functioning and well-being of patients with FSHD. Rather than replace the clinical outcome scales used in clinical drug development, these at home measurements using wearable devices, generating real-world data, may turn out to be essential complementary tools in the very near future.
Interested to learn more? Visit Trial@home or reach out to info@chdr.nl.
