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

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 ...

Source: Orphanet Journal of Rare Diseases - April 29, 2019 Category: Internal Medicine Authors: Magdalena Harrington, Diane Whalley, James Twiss, Rebecca Rushton, Susan Martin, Lynn Huynh and Hongbo Yang Tags: Research Source Type: research