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

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 ...

Source: Orphanet Journal of Rare Diseases - February 27, 2021 Category: Internal Medicine Authors: Bethanie Garside, Jan Hoong Ho, See Kwok, Yifen Liu, Shaishav Dhage, Rachelle Donn, Zohaib Iqbal, Simon A. Jones and Handrean Soran Tags: Research Source Type: research