Preliminary development of proxy-rated quality-of-life scales for children and adults with Niemann-Pick type C.
Lydia AstonRachel ShawRebecca C KnibbPublished in: Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation (2019)
The NPCQLQ-C and NPCQLQ-A are the first disease-specific QoL scales to be developed for people living with NPC. This novel approach to scale development values the experiential, real life impact of living with NPC and focused on the lived-experiences and impact on QoL. The scales will enable healthcare professionals and researchers to have a better understanding and quantifiable measurement of the impact of living with NPC on a patient's daily life.