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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...
Autores principales: | , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2019
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Materias: | |
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 |