0000000001189755

AUTHOR

H. Soran

showing 2 related works from this author

The role of registries in rare genetic lipid disorders: Review and introduction of the first global registry in lipoprotein lipase deficiency

2017

International audience; A good understanding of the natural history of rare genetic lipid disorders is a pre-requisite for successful patient management. Disease registries have been helpful in this regard. Lipoprotein Lipase Deficiency (LPLD) is a rare, autosomal-recessive lipid disorder characterized by severe hypertriglyceridemia and a very high risk for recurrent acute pancreatitis, however, only limited data are available on its natural course. Alipogene tiparvovec (Glybera (R)) is the first gene therapy to receive Marketing Authorization in the European Union; GENIALL (GENetherapy In the MAnagement of Lipoprotein Lipase Deficiency), a 15-year registry focusing on LPLD was launched in …

0301 basic medicinePediatricsPathologySettore MED/09 - Medicina Interna[SDV]Life Sciences [q-bio]Familial hypercholesterolemiaDisease030204 cardiovascular system & hematologyGeneTHERAPY0302 clinical medicineFamilialRisk FactorsHyperchylomicronemiaAlipogene tiparvovecRegistriesFAMILIAL HYPERCHOLESTEROLEMIAmedia_commonHypertriglyceridemiaPrognosis3. Good healthNatural historySystematic reviewPhenotypeDISEASESSAFETYHyperlipoproteinemia Type ICardiology and Cardiovascular Medicinemedicine.medical_specialtyAPHERESISRegistryFamilial chylomicronemia syndromeGENIALLLysosomal acid lipase deficiencyLipid Metabolism Inborn Errors03 medical and health sciencesLipoprotein lipase deficiencyRare DiseasesGene therapychylomicronemia syndromemedicinemedia_common.cataloged_instanceHumansGenetic Predisposition to DiseaseEuropean unionLipoprotein lipase deficiency (LPLD)business.industryALIPOGENE TIPARVOVEC AAV1-LPLS447Xmedicine.diseaseAlipogene tiparvovecLipoprotein Lipase030104 developmental biologyOrphan diseasebusiness
researchProduct

Cardiovascular Efficacy and Safety of Bococizumab in High-Risk Patients

2017

Item does not contain fulltext BACKGROUND: Bococizumab is a humanized monoclonal antibody that inhibits proprotein convertase subtilisin-kexin type 9 (PCSK9) and reduces levels of low-density lipoprotein (LDL) cholesterol. We sought to evaluate the efficacy of bococizumab in patients at high cardiovascular risk. METHODS: In two parallel, multinational trials with different entry criteria for LDL cholesterol levels, we randomly assigned the 27,438 patients in the combined trials to receive bococizumab (at a dose of 150 mg) subcutaneously every 2 weeks or placebo. The primary end point was nonfatal myocardial infarction, nonfatal stroke, hospitalization for unstable angina requiring urgent re…

MaleSTATIN THERAPYAnticholesteremic Agents/adverse effectsAntibodieVascular damage Radboud Institute for Health Sciences [Radboudumc 16]Injections Subcutaneous/adverse effects030204 cardiovascular system & hematologyBococizumablaw.inventionPCSK90302 clinical medicineRandomized controlled triallawRisk FactorsGENETIC-VARIANTSCardiovascular DiseaseMonoclonalAnticholesteremic Agent030212 general & internal medicineMyocardial infarctionTreatment FailureHumanizedProprotein Convertase 9/antagonists & inhibitorsMedicine(all)Antibodies; Antibodies Monoclonal Humanized; Anticholesteremic Agents; Cardiovascular Diseases; Cholesterol LDL; Double-Blind Method; Female; Follow-Up Studies; Humans; Hypercholesterolemia; Injections Subcutaneous; Lipids; Male; Middle Aged; Proprotein Convertase 9; Risk Factors; Treatment Failure; Medicine (all)Anticholesteremic AgentsMedicine (all)PCSK9 InhibitorsAntibodies; antibodies monoclonal humanized; anticholesteremic agents; cardiovascular diseases; cholesterol LDL; double-blind method; female; follow-up studies; humans; hypercholesterolemia; injections subcutaneous; lipids; male; middle aged; proprotein convertase 9; risk factors; treatment failure; medicine (all)Vascular damage Radboud Institute for Molecular Life Sciences [Radboudumc 16]General MedicineLipidMiddle AgedLipids3. Good healthLDL/bloodMulticenter StudyCholesterolTRIALSCholesterol LDL/bloodCardiovascular DiseasesAntibodies Monoclonal Humanized/adverse effectsanticholesteremic agentsRandomized Controlled Trialsubcutaneouslipids (amino acids peptides and proteins)FemaleProprotein Convertase 9Cardiovascular Diseases/prevention & controlREDUCING LIPIDSHumanmedicine.medical_specialtyanimal structuresInjections SubcutaneousHypercholesterolemiaHypercholesterolemia/drug therapyPlaceboAntibodies Monoclonal HumanizedInjections SubcutaneouAntibodiesLDLInjectionsFollow-Up StudielipidsEVENTS03 medical and health sciencesantibodies monoclonal humanizedDouble-Blind MethodInternal medicinemedicineJournal ArticleHumansComparative StudyMETAANALYSISAlirocumabbusiness.industryUnstable anginaLipids/bloodPCSK9Risk FactorfungiAntibodies/bloodCholesterol LDLta3121medicine.diseaseSurgerycardiovascular diseasesEvolocumabREDUCTIONHumanized/adverse effectsSubcutaneous/adverse effectsbusiness[SDV.MHEP]Life Sciences [q-bio]/Human health and pathologyFollow-Up Studies
researchProduct