ESPE Yearbook of Paediatric Endocrinology

ESPE Yearbook of Paediatric Endocrinology

ISSN 1662-4009 (online)

Published by BioScientifica

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ey0021.12-16 | Lipid Metabolism | ESPEYB21

12.16. Evinacumab for pediatric patients with homozygous familial hypercholesterolemia

A Wiegman , S Greber-Platzer , S Ali , MD Reijman , EA Brinton , MJ Charng , S Srinivasan , C Baker-Smith , S Baum , JA Brothers , J Hartz , PM Moriarty , J Mendell , S Bihorel , P Banerjee , RT George , B Hirshberg , R Pordy

Homozygous familial hypercholesterolemia (HoFH) is a severe disorder caused by genetic mutations in LDLR (encoding the LDL receptor), APOB or PCSK9. LDL-C levels in HoFH are extremely elevated)>400 mg/dL(even in utero, leading to cardiovascular events, and disability or death during childhood and adolescence. Conventional medications have minimal efficacy, since LDL-C levels cannot be reduced through upregulation of hepatic LDL receptors.<p c...
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Published by BioScientifica

ESPE Yearbook of Paediatric Endocrinology
ISSN 1662-4009 (Online)
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