Changes in PCSK 9 and apolipoprotein B100 in Niemann-Pick disease after enzyme replacement therapy with olipudase alfa.
Bethanie GarsideJan Hoong HoSee KwokYifen LiuShaishav DhageRachelle DonnZohaib IqbalSimon A JonesHandrean SoranPublished in: Orphanet journal of rare diseases (2021)
This study demonstrated that patients with NPD-B had a proatherogenic lipid profile and higher circulating TNF-α compared to reference group. There was an improvement in dyslipidaemia after olipudase alfa. It was possible that reductions in LDL-C and apoB100 were driven by reductions in TNF-α and PCSK9 following ERT.