Search results for "Inclisiran"

showing 4 items of 4 documents

Inclisiran: a small interfering RNA strategy targeting PCSK9 to treat hypercholesterolemia

2022

Introduction: Inclisiran is a novel posttranscriptional gene silencing therapy that inhibits proprotein convertase subtilisin/kexin type 9 (PCSK9) synthesis by RNA interference and has a potent, dose-dependent, durable effect in lowering LDL-C, and therefore is an effective drug to treat dyslipidemia, reducing the risk for acute cardiovascular (CV) events. It is safe and well-tolerated. Areas covered: This paper aims to review the mechanism of action of inclisiran while evaluating its efficacy and safety in the treatment of dyslipidemia from data of the clinical trials in the ORION program. Expert opinion: Data from the clinical trials in the ORION program demonstrated efficacy and safety o…

DrugSmall interfering RNAmedia_common.quotation_subjectHypercholesterolemiaPlaceboBioinformaticsLDLPCSK9RNA interferencemedicineAnimalsHumansPharmacology (medical)Gene SilencingRNA Small InterferingAdverse effectDyslipidemiasmedia_commontherapy.business.industryPCSK9General Medicinemedicine.diseaseClinical trialCardiovascular DiseasesAtherosclerosis inclisiran LDL PCSK9 RNA therapy Animals Cardiovascular Diseases Dyslipidemias Gene Silencing Humans Hypercholesterolemia Proprotein Convertase 9 RNA Small InterferingAtherosclerosiRNAProprotein Convertase 9businessinclisiranDyslipidemiaExpert Opinion on Drug Safety
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Insight into the Evolving Role of PCSK9

2022

Proprotein convertase subtilisin/kexin type 9 (PCSK9) is the last discovered member of the family of proprotein convertases (PCs), mainly synthetized in hepatic cells. This serine protease plays a pivotal role in the reduction of the number of low-density lipoprotein receptors (LDLRs) on the surface of hepatocytes, which leads to an increase in the level of cholesterol in the blood. This mechanism and the fact that gain of function (GOF) mutations in PCSK9 are responsible for causing familial hypercholesterolemia whereas loss-of-function (LOF) mutations are associated with hypocholesterolemia, prompted the invention of drugs that block PCSK9 action. The high efficiency of PCSK9 inhibitors (…

Endocrinology Diabetes and MetabolismPCSK9; cholesterol; anti-PCSK9; cardiovascular diseases; genetic; atherosclerosis; alirocumab; evolocumab; inclisiranMolecular BiologyBiochemistryMetabolites
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New Frontiers in the Treatment of Homozygous Familial Hypercholesterolemia.

2021

: Homozygous familial hypercholesterolemia (HoFH) is a rare genetic disorder. The most common cause is a mutation in both alleles of the gene encoding for the low-density lipoprotein (LDL) receptor, although other causative mutations have been identified. Complications of atherosclerotic cardiovascular disease are common in these patients; therefore, reducing the elevated LDL-cholesterol burden is critical in their management. Conventionally, this is achieved by patients initiating lipid-lowering therapy, but this can present challenges in clinical practice. Fortunately, novel therapeutic strategies have enabled promising innovations in HoFH treatment. This review highlights recent and ongo…

Settore MED/09 - Medicina InternaGenetic enhancementHomozygous familial hypercholesterolemiaFamilial hypercholesterolemiaInclisiranBioinformaticsmedicine.disease_causeBenzimidazolePCSK9Hyperlipoproteinemia Type IIchemistry.chemical_compoundGene therapyAnticholesteremic AgentmedicineAngiopoietin-like 3HumansLow-density lipoprotein cholesterolAlleleAngiopoietin-like 3; Gene therapy; Gene-editing; Homozygous familial hypercholesterolemia; Inclisiran; Lomitapide; Low-density lipoprotein cholesterol; PCSK9MutationGene-editingAtherosclerotic cardiovascular diseasebusiness.industryPCSK9Anticholesteremic AgentsHomozygoteGenetic disorderGeneral MedicineCholesterol LDLmedicine.diseaseLomitapideLomitapidechemistrylipids (amino acids peptides and proteins)BenzimidazolesCardiology and Cardiovascular MedicinebusinessHumanHeart failure clinics
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Which patients at risk of cardiovascular disease might benefit the most from inclisiran? – The expert opinion of the Polish experts. The compromise b…

2022

It is the statement of the Polish Experts on the group of patients that might benefit the most from inclisiran. We indicated the fastest way to have inclisiran available for the polish patients, with the necessary changes of the existing drug program for PCSK9 inhibitors (B-101), explaining why it is the optimal way, and why, taking into account available EBM data (the ORION program), inclisiran should be added to this program. We also present some perspective on the future necessary changes in the availability of the innovative therapies such us PCSK9 targeted therapy, what, taking into account the effectiveness of LDL-C goal achievement in Poland for very high CVD risk patients (only 17%)…

drug programlipid lowering therapyhigh risk patientsGeneral MedicinePolandinclisiranreimbursementArchives of Medical Science
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