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metachromatic leukodystrophy · Vinony

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EntityQ1120682· pop 23· linked from 135 articles

metachromatic leukodystrophy

human disease

Research

1,902 papers

Metachromatic leukodystrophy: To screen or not to screen?European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society · 2023
Mutation Update of ARSA and PSAP Genes Causing Metachromatic Leukodystrophy.Human mutation · 2016
Atidarsagene autotemcel for metachromatic leukodystrophy.Drugs of today (Barcelona, Spain : 1998) · 2023
Metachromatic leukodystrophy--an update.Neuropediatrics · 2010
Metachromatic leukodystrophy: A story of hope woven from sorrow.Molecular therapy : the journal of the American Society of Gene Therapy · 2024

Connections

hematopoietic stem cell transplantation

Categories

Autosomal recessive disorders Demyelinating diseases of CNS Leukodystrophies Lipid storage disorders Neurological disorders in children Rare diseases

Sources

wikipedia.externallinksexternallink

myelin

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