Understanding Symptoms in RYR1-Related Myopathies: A Mixed-Methods Analysis Based on Participants' Experience.

Carlos Capella-Peris Mary M Cosgrove Irene C Chrismer M Sonia RazaqyarJeffrey S ElliottAnna KuoMagalie Emile-BackerKatherine G Meilleur

Published in: The patient (2021)

We present a comprehensive characterization of symptoms in RYR1-RM and how those symptoms influence HRQoL. Furthermore, the introduction of mixed methods may be a valuable way to better understand patient-centered data in rare diseases to support affected individuals in coping with their symptoms.

Keyphrases

sleep quality
study protocol
depressive symptoms
clinical trial
big data
social support
machine learning
deep learning