Assessing the multidimensional burden of facioscapulohumeral muscular dystrophy through patient-reported outcomes and experience
Wenjing Ji·Yu Fang·Qifang He·Linkai Xu·MinTing Lin·Linguo Li·Minghui Zeng·Tingting Liu·Zhiqiang Wang·Yifei Zhao·Ailiyaer Aikepaier·Yuhua Lin·Zikai He·Huxinigeer Maimaiti·Yuqiang Kang·Xinyue Chen·Sundus Shukar
The findings highlight a substantial disease burden and significant unmet needs among FSHD patients. Early diagnosis, approval of targeted therapies, and comprehensive support from families, healthcare systems, and society are imperative. These measures are essential to improve health outcomes and quality of life for this patient population.