Article

The Burden of Disease in Metachromatic Leukodystrophy: Results of a Caregiver Survey in the UK and Republic of Ireland.

2023-05-04

Abstract excerpt

<title>Abstract</title> <p>Background: Metachromatic Leukodystrophy (MLD) is a rare, autosomal recessive lysosomal storage disease characterised by the progressive loss of motor function and severe decline in cognitive function. Palliative care is the only option for most patients with the disease eventually leading to premature death. Three UK-based MLD patient organisations commissioned an online survey, and fo...

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Literature Corpus work: 8a7fc4c0-f00f-5e58-8ee3-43086c3d6e74
DOI: 10.21203/rs.3.rs-2271864/v1

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