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Clearance of Hepatic Sphingomyelin by Olipudase Alfa Is Associated With Improvement in Lipid Profiles in Acid Sphingomyelinase Deficiency

Acid sphingomyelinase deficiency (ASMD; Niemann-Pick disease type A and B) is a lysosomal storage disorder characterized by abnormal intracellular sphingomyelin (SM) accumulation. Prominent liver involvement results in hepatomegaly, fibrosis/cirrhosis, abnormal liver chemistries, and a proatherogeni...

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Detalles Bibliográficos
Autores principales:	Thurberg, Beth L., Wasserstein, Melissa P., Jones, Simon A., Schiano, Thomas D., Cox, Gerald F., Puga, Ana Cristina
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Wolters Kluwer Health, Inc 2016
Materias:	Original Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4987207/ https://www.ncbi.nlm.nih.gov/pubmed/27340749 http://dx.doi.org/10.1097/PAS.0000000000000659

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