Changes in PCSK 9 and apolipoprotein B100 in Niemann–Pick disease after enzyme replacement therapy with olipudase alfa

Changes in PCSK 9 and apolipoprotein B100 in Niemann–Pick disease after enzyme replacement therapy with olipudase alfa

Abstract Background Enzyme replacement therapy (ERT) with olipudase alfa, a recombinant human acid sphingomyelinase (rhASM), is being developed to treat patients with ASM deficiency (ASMD), commonly known as Niemann–Pick disease (NPD) types A or B. This study assessed the effect of ERT on lipid para...

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Bibliographic Details
Main Authors: Bethanie Garside, Jan Hoong Ho, See Kwok, Yifen Liu, Shaishav Dhage, Rachelle Donn, Zohaib Iqbal, Simon A. Jones, Handrean Soran
Format: Article
Language:English
Published: BMC 2021-02-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Acid sphingomyelinase deficiency
Apolipoprotein B100
Proprotein convertase subtilisin/klexin type 9
Tumour necrosis factor α
Low density lipoprotein cholesterol
Enzyme replacement therapy
Online Access:https://doi.org/10.1186/s13023-021-01739-y

Internet

https://doi.org/10.1186/s13023-021-01739-y

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