Disease Severity And Motor Impairment Correlate With Health-Related Quality Of Life In Ap-4-Associated Hereditary Spastic Paraplegia
ANNALS OF NEUROLOGY(2021)
摘要
We show that the CPCHILD questionnaire, developed for use in children with cerebral palsy, can be used to assess HRQoL in patients with childhood-onset complex hereditary spastic paraplegia. HRQoL is reduced in patients with AP-4-HSP and correlates with the degree of motor impairment. These results provide a framework for medical decision making and a baseline for the future development of treatment guidelines and interventional trials.
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关键词
Movement Disorders (including Cerebral Palsy), Genetics, Rare Diseases
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