Article

An International Study of Caregiver-Reported Burden and Quality of Life in Metachromatic Leukodystrophy

2022-05-03

Abstract excerpt

<title>Abstract</title> <p><bold>Background: </bold>Metachromatic leukodystrophy (MLD) is an autosomal recessive lysosomal disorder caused by mutations in the arylsulfatase A gene. Until now, there has been little information on the burden of MLD on patients and their caregivers. This multinational study aims to quantify caregiver-related impacts of MLD across several key domains including symptoms, treatment bur...

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Literature Corpus work: 90a85457-af94-55e0-bb36-f4a6a4d029f0
DOI: 10.21203/rs.3.rs-1559430/v1

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