Inclisiran Durably Lowers Low-Density Lipoprotein Cholesterol and Proprotein Convertase Subtilisin/Kexin Type 9 Expression in Homozygous Familial Hypercholesterolemia: The ORION-2 Pilot Study

Circulation. 2020 Jun 2;141(22):1829-1831. doi: 10.1161/CIRCULATIONAHA.119.044431. Epub 2020 Jun 1.

Authors

G Kees Hovingh¹, Norman E Lepor², David Kallend³, Robert M Stoekenbroek³, Peter L J Wijngaard³, Frederick J Raal⁴

Affiliations

¹ Department of Internal Medicine, Amsterdam UMC, The Netherlands (G.K.H.).
² Geffen School of Medicine, University of California, Los Angeles (N.E.L.).
³ Cardiovascular, The Medicines Company, Parsippany, NJ (D.K., R.M.S., P.L.J.W.).
⁴ Department of Medicine, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa (F.J.R.).

PMID: 32479195
DOI: 10.1161/CIRCULATIONAHA.119.044431

No abstract available

Keywords: ALN-PCS; cholesterol, LDL; hypercholesterolemia.

Publication types

Letter
Multicenter Study
Research Support, Non-U.S. Gov't

MeSH terms

Anticholesteremic Agents / therapeutic use
Cholesterol, LDL / biosynthesis*
Ezetimibe / therapeutic use
Gene Knockdown Techniques
Genetic Therapy*
Homozygote
Humans
Hydroxymethylglutaryl-CoA Reductase Inhibitors / therapeutic use
Hyperlipoproteinemia Type II / drug therapy
Hyperlipoproteinemia Type II / genetics
Hyperlipoproteinemia Type II / metabolism
Hyperlipoproteinemia Type II / therapy*
Molecular Targeted Therapy*
PCSK9 Inhibitors*
Pilot Projects
Proof of Concept Study
Proprotein Convertase 9 / biosynthesis
Proprotein Convertase 9 / genetics
RNA Interference*
RNA, Small Interfering / adverse effects
RNA, Small Interfering / genetics
RNA, Small Interfering / pharmacology
RNA, Small Interfering / therapeutic use*
Treatment Outcome

Substances

ALN-PCS
Anticholesteremic Agents
Cholesterol, LDL
Hydroxymethylglutaryl-CoA Reductase Inhibitors
PCSK9 Inhibitors
RNA, Small Interfering
PCSK9 protein, human
Proprotein Convertase 9
Ezetimibe