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Lomitapide for the treatment of paediatric patients with homozygous familial hypercholesterolaemia (APH-19): results from the efficacy phase of an open-label, multicentre, phase 3 study

L. Masana, Zambon Alberto, Claus Peter Schmitt, Christina Taylan, Joenna Driemeyer, Hofit Cohen, Paola Sabrina Buonuomo, Abdullah Al‐Ashwal, Mohammed Al-Dubayee, Naji Kholaif, José Luis Díaz-Díaz, Faouzi Maatouk, Sergio Martínez‐Hervás, Brian Mangal, Sandra Löwe, Tracy Cunningham

2024The Lancet Diabetes & Endocrinology26 citationsDOI

Topics & Concepts

MedicinePhase (matter)Open labelPediatricsInternal medicineIntensive care medicineClinical trialChemistryOrganic chemistryLipoproteins and Cardiovascular HealthDiabetes, Cardiovascular Risks, and LipoproteinsCholesterol and Lipid Metabolism
Lomitapide for the treatment of paediatric patients with homozygous familial hypercholesterolaemia (APH-19): results from the efficacy phase of an open-label, multicentre, phase 3 study | Litcius