Efficacy of Long-Term Treatment of Autosomal Recessive Hypercholesterolemia With Lomitapide: A Subanalysis of the Pan-European Lomitapide Study

Efficacy of Long-Term Treatment of Autosomal Recessive Hypercholesterolemia With Lomitapide: A Subanalysis of the Pan-European Lomitapide Study

Backgroundand aim: Autosomal recessive hypercholesterolemia (ARH) is a rare autosomal recessive disorder of low-density lipoprotein (LDL) metabolism caused by pathogenic variants in the LDLRAP1 gene. Like homozygous familial hypercholesterolemia, ARH is resistant to conventional LDL-lowering medicat...

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Main Authors: Laura D’Erasmo, Antonina Giammanco, Patrizia Suppressa, Chiara Pavanello, Gabriella Iannuzzo, Alessia Di Costanzo, Daniele Tramontano, Ilenia Minicocci, Simone Bini, Anja Vogt, Kim Stewards, Jeanine Roeters Van Lennep, Stefano Bertolini, Marcello Arca, the Italian and European Working Group on Lomitapide in HoFH, Maurizio Averna, Eric Boersma, Katia Bonomo, Marco Bucci, Laura Calabresi, Paolo Calabrò, Angelo Baldassare Cefalù, Jaimini Cegla, Arturo Cesaro, Sergio D’Addato, Eugene Daphnis, Maria Donata Di Taranto, Avishay Ellis, Fabio Fimiani, Giuliana Fortunato, Marco Gentile, Meral Kayikcioglu, Genovefa Kolovou, Evangelos Liberopoulos, Karin Littmann, Sergio Martínez-Hervás, Tiziana Montalcini, Fabio Nota, Livia Pisciotta, Arturo Puja, Giovanni José Real, Jeanine Roeters van Lennep, Joost Rutten, Carlo Sabbà, Tiziana Sampietro, Francesco Sbrana, Kim Steward, Fulvio Ventura, Battista Vigna, Shahenaz Walji
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-08-01
Series:Frontiers in Genetics
Subjects:
Real-world study
rare disease
autosomal recessive hypercholesterolaemia
LDL-C
lomitapide
long-term
Online Access:https://www.frontiersin.org/articles/10.3389/fgene.2022.937750/full

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https://www.frontiersin.org/articles/10.3389/fgene.2022.937750/full

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