Prenusinersen economic and health-related quality of life burden of spinal muscular atrophy.
Georgina M ChambersStella Nalukwago SettumbaKate A CareyAnita CairnsManoj P MenezesMonique RyanMichelle A FarrarPublished in: Neurology (2020)
Our results demonstrate the substantial and far-ranging economic and quality of life burden on households and society of SMA and are essential to fully understanding the health benefits and cost-effectiveness associated with emerging disease-modifying therapies for SMA.