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Article

Efficacy of Rosuvastatin in Children With Homozygous Familial Hypercholesterolemia and Association With Underlying Genetic Mutations.

Journal of the American College of Cardiology - 29 Aug 2017

Stein Evan A, Dann Eldad J, Wiegman Albert, Skovby Flemming, Gaudet Daniel, Sokal Etienne, Charng Min-Ji, Mohamed Mafauzy, Luirink Ilse, Raichlen Joel S, Sundén Mattias, Carlsson Stefan C, Raal Frederick J, Kastelein John J P

Abstract excerpt

BACKGROUND: Homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder, is characterized by extremely elevated levels of low-density lipoprotein cholesterol (LDL-C) and accelerated atherosclerotic cardiovascular disease. Statin treatment starts at diagnosis, but no statin has been formally evaluated in, or approved for, HoFH children. OBJECTIVES: The authors sought to assess the LDL-C efficacy of...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 28838366
DOI: 10.1016/j.jacc.2017.06.058

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026. Manifest publication-graph-cohort-2026-09-05.1.

Source receipt: pubmed, retrieved 5 Sept 2026.

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