Insights into the natural history of metachromatic leukodystrophy from interviews with caregivers
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DOI: 10.1186/s13023-019-1060-2
Abstract: Background and methodsMetachromatic 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… read more here.
Keywords: juvenile mld; disease; natural history; late infantile ... See more keywords