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

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 disease rema...

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Detalles Bibliográficos
Autores principales: Harrington, Magdalena, Whalley, Diane, Twiss, James, Rushton, Rebecca, Martin, Susan, Huynh, Lynn, Yang, Hongbo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2019
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6489348/
https://www.ncbi.nlm.nih.gov/pubmed/31036045
http://dx.doi.org/10.1186/s13023-019-1060-2

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