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

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 parameters a...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Garside, Bethanie, Ho, Jan Hoong, Kwok, See, Liu, Yifen, Dhage, Shaishav, Donn, Rachelle, Iqbal, Zohaib, Jones, Simon A., Soran, Handrean
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2021
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7913414/
https://ncbi.nlm.nih.gov/pubmed/33639994
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-021-01739-y
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