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Lysosomal Acid Lipase Deficiency: Therapeutic Options

Lysosomal acid lipase (LAL) deficiency is a metabolic (storage) disorder, encompassing a severe (Wolman disease) and attenuated (Cholesterol ester storage disease) subtype; both inherited as autosomal recessive traits. Cardinal clinical features include the combination of hepatic dysfunction and dys...

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Bibliografiske detaljer
Udgivet i:Drug Des Devel Ther
Main Authors: Pastores, Gregory M, Hughes, Derralynn A
Format: Artigo
Sprog:Inglês
Udgivet: Dove 2020
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7023879/
https://ncbi.nlm.nih.gov/pubmed/32103901
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/DDDT.S149264
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