Discovery of Lomitapide as a Novel Mast Cell Regulatory Agent Through Chemical Library Screening. [PDF]
Eur J ImmunolAllergic diseases are a global health concern, and new molecular insights and therapeutic agents are necessary to support future research and interventions.
Mamorita S +3 more
europepmc +3 more sources
Orphanet Journal of Rare DiseasesBackground We assessed long-term real-world effectiveness and safety of lomitapide in patients with homozygous familial hypercholesterolemia (HoFH). Methods Retrospective case series of six patients with HoFH treated with lomitapide in an Italian clinic.
Patrizia Suppressa +3 more
doaj +3 more sources
Efficacy of Long-Term Treatment of Autosomal Recessive Hypercholesterolemia With Lomitapide: A Subanalysis of the Pan-European Lomitapide Study [PDF]
Frontiers in Genetics, 2022 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.
Laura D’Erasmo +59 more
doaj +8 more sources
Binding of Selected Ligands to Human Protein Disulfide Isomerase and Microsomal Triglyceride Transfer Protein Complex and the Associated Conformational Changes: A Computational Molecular Modelling Study [PDF]
ChemistryOpenHuman protein disulfide isomerase (PDI) is a multifunctional protein, and also serves as the β subunit of the human microsomal triglyceride transfer protein (MTP) complex, a lipid transfer machinery.
Yong Xiao Yang, Peng Li, Bao Ting Zhu
doaj +3 more sources
RFWD3 Reprograms Nucleotide Metabolism Through PHGDH to Induce Chemoresistance In Osteosarcoma [PDF]
Advanced ScienceChemoresistance represents a major challenge for osteosarcoma treatment. Despite the improved knowledge of cancer biology, the core determinants of cisplatin (DDP) resistance in osteosarcoma remain unclear and deserve further exploration.
Wenchao Zhang +6 more
doaj +3 more sources
Lomitapide en hipercolesterolemia familiar heterocigota [PDF]
Revista Colombiana de Endocrinología, Diabetes y Metabolismo, 2022 Introducción: la hipercolesterolemia familiar (HF) es una enfermedad autosómica dominante causada por mutaciones del gen que codifica el receptor de colesterol de baja densidad (LDLR) o en moléculas asociadas al funcionamiento de este.
Juan Esteban Bedoya Loaiza +2 more
doaj +2 more sources
The Beneficial Effect of Lomitapide on the Cardiovascular System in LDLr−/− Mice with Obesity
Antioxidants, 2023 Objectives: Homozygous familial hypercholesteremia (HoFH) is a rare, life-threatening metabolic disease, mainly caused by a mutation in the LDL receptor. If untreated, HoFH causes premature death from acute coronary syndrome.
Undral Munkhsaikhan +8 more
doaj +2 more sources
Cell Death and Disease, 2022 Autophagy is a biological process that maintains cellular homeostasis and regulates the internal cellular environment. Hyperactivating autophagy to trigger cell death has been a suggested therapeutic strategy for cancer treatment.
Boah Lee +9 more
doaj +2 more sources
Long-term efficacy of lipoprotein apheresis and lomitapide in the treatment of homozygous familial hypercholesterolemia (HoFH): a cross-national retrospective survey [PDF]
Orphanet Journal of Rare Diseases, 2021 Background Homozygous familial hypercholesterolemia (HoFH) is a rare life-threatening condition that represents a therapeutic challenge. The vast majority of HoFH patients fail to achieve LDL-C targets when treated with the standard protocol, which ...
Laura D’Erasmo +34 more
doaj +2 more sources
Treatment patterns in homozygous familial hypercholesterolemia: Undertreatment and delayed adoption of low-density lipoprotein receptor–independent therapies [PDF]
American Journal of Preventive CardiologyBackground: Homozygous familial hypercholesterolemia (HoFH) is an ultrarare disease characterized by extremely high levels of low-density lipoprotein cholesterol (LDL-C) and low-density lipoprotein receptor (LDLR) dysfunction, making most LDLR function ...
Dana J. Murdock +5 more
doaj +2 more sources