Estimation Of The Quality Of Life Benefits Associated With Treatment For Spinal Muscular Atrophy.

Andrew J LloydRobin ThompsonKaty GallopMegan Teynor

Published in: ClinicoEconomics and outcomes research : CEOR (2019)

The utility scores obtained in this study highlight the very substantial burden experienced by SMA patients. Despite the limitations in the methods used, this study produced data with face validity and is a useful starting point for understanding the burden of SMA Types I and II in cost-effectiveness analysis.

Keyphrases

end stage renal disease
newly diagnosed
ejection fraction
chronic kidney disease
risk factors
prognostic factors
machine learning
peritoneal dialysis
combination therapy
patient reported outcomes