Disease Burden and Living Situation of Patients With Facioscapulohumeral Muscular Dystrophy

The goal of this observational study is to learn the patient journey, disease burden, living situation, quality of life and etc. in patients with Facioscapulohumeral Muscular Dystrophy in all sex/gender and age groups. The main questions it aims to answer are: 1. What's the patient journey of patients with Facioscapulohumeral Muscular Dystrophy? 2. How does Facioscapulohumeral Muscular Dystrophy burden the patients? 3. How's the quality of life of patients with Facioscapulohumeral Muscular Dystrophy? Patients with Facioscapulohumeral Muscular Dystrophy will be asked to: 1. fill a questionnaire. 2. be interviewed and answer questions about their illness experience, economic burden, quality of life and etc. Medical experts on Facioscapulohumeral Muscular Dystrophy will be asked to be interviewed and answer questions about understanding on the disease and the patients.

NCT Number	NCT06517498
Lead Sponsor	Xi'an Jiaotong University Collaborators: Chinese Organization for Rare Disorders, First Affiliated Hospital of Fujian Medical University
Conditions	Muscular Dystrophy, Facioscapulohumeral
Enrollment	300 participants
Start Date	2024-03-01
Primary Completion	2025-03 (estimated)
Study Completion	2025-07 (estimated)
Updated on ClinicalTrials.gov	2024-07-24