Preliminary development of proxy-rated quality-of-life scales for children and adults with Niemann-Pick type C.

Objectives: Niemann-Pick disease type C (NPC) is a rare life-limiting disease for which there is no cure. No scales currently exist to measure the impact of medication, physical therapy or clinical trials. The aim of this study was to develop age-appropriate Quality-of-Life (QoL) scales to measure t...

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Detalles Bibliográficos
Publicado en:Quality of Life Research Vol. 28; no. 11; pp. 3083 - 3093
Autores principales: Aston, Lydia, Shaw, Rachel, Knibb, Rebecca
Formato: research tables/charts Journal Article
Publicado: Springer Nature Nov2019
Acceso en línea:Ver este registro en EBSCOhost