Abstract
Metachromatic leukodystrophy (MLD) is a rare lysosomal storage disease caused by deficient arylsulfatase A activity, leading to progressive cognitive and motor dysfunction and death. We estimated late-infantile MLD (LI-MLD) patient and caregiver health state utilities to inform cost–utility evaluations of MLD treatments. Utilities range from 1 (full health) to 0 (dead); values <0 reflect states considered worse than dead.
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