Insights into the natural history of metachromatic leukodystrophy from interviews with caregivers

Insights into the natural history of metachromatic leukodystrophy from interviews with caregivers

Abstract Background and methods Metachromatic leukodystrophy (MLD) is a rare, autosomal recessive lysosomal storage disease caused by deficient activity of arylsulfatase A. Neurological involvement results in severe disability and premature death, but understanding of the natural history of the dise...

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Bibliographic Details
Main Authors: Magdalena Harrington, Diane Whalley, James Twiss, Rebecca Rushton, Susan Martin, Lynn Huynh, Hongbo Yang
Format: Article
Language:English
Published: BMC 2019-04-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Metachromatic leukodystrophy
MLD
Lysosomal storage disease
Caregiver
Qualitative research
Natural history
Online Access:http://link.springer.com/article/10.1186/s13023-019-1060-2

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http://link.springer.com/article/10.1186/s13023-019-1060-2

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