2022
Designing ATHN 7: Safety, effectiveness and practice of treatment of people with haemophilia in the United States through a natural history cohort study
Buckner T, Daoud N, Miles M, O'Neill C, Watson C, Recht M. Designing ATHN 7: Safety, effectiveness and practice of treatment of people with haemophilia in the United States through a natural history cohort study. The Journal Of Haemophilia Practice 2022, 9: 147-155. DOI: 10.2478/jhp-2022-0020.Peer-Reviewed Original ResearchEuropean Haemophilia Safety SurveillanceNatural history cohort studyHemophilia therapyCohort studyPatient-reported outcomesBleeding rateDevelopment of inhibitory antibodiesFactor VIII and IXAnnualised bleeding rateIncidence of safety eventsPrevention of bleedingObservational cohort studyRecombinant clotting factorsPrimary outcome measureTime of enrollmentInhibitory antibodiesTRANSCEND studiesClotting factorsHemophiliaTherapyPractice of treatmentAmerican ThrombosisOutcome measuresTreatment of peopleRegulatory approval
2019
The national blueprint for 21st century data and specimen collection and observational cohort studies: NHLBI State of the Science Workshop on factor VIII inhibitors
Konkle B, Recht M, Group T. The national blueprint for 21st century data and specimen collection and observational cohort studies: NHLBI State of the Science Workshop on factor VIII inhibitors. Haemophilia 2019, 25: 590-594. PMID: 31329362, DOI: 10.1111/hae.13772.Peer-Reviewed Original ResearchConceptsFactor VIII inhibitorHemophilia AFactor VIIIDeficiency of coagulation factor VIIIDevelopment of inhibitory antibodiesSpecimen collectionComplication of haemophilia AObservational cohort studyPublic health impactCoagulation factor VIIIPatient/family engagementNational blueprintFVIII concentratesCongenital deficiencyInhibitory antibodiesNational HeartCohort studyGroup 2Patient populationSevere formTraining opportunitiesPatientsHealth impactsMedical systemComplications
2013
An Inhibitory Antibody Blocks the First Step in the Dithiol/Disulfide Relay Mechanism of the Enzyme QSOX1
Grossman I, Alon A, Ilani T, Fass D. An Inhibitory Antibody Blocks the First Step in the Dithiol/Disulfide Relay Mechanism of the Enzyme QSOX1. Journal Of Molecular Biology 2013, 425: 4366-4378. PMID: 23867277, DOI: 10.1016/j.jmb.2013.07.011.Peer-Reviewed Original ResearchMeSH KeywordsAmino Acid SequenceAntibodies, BlockingAntibodies, MonoclonalBinding SitesCell AdhesionCell MovementDisulfidesHumansKineticsModels, MolecularMolecular Sequence DataMultiprotein ComplexesOxidoreductases Acting on Sulfur Group DonorsProtein BindingProtein ConformationProtein Interaction Domains and MotifsSingle-Chain AntibodiesTolueneConceptsQuiescin sulfhydryl oxidase 1Catalyst of disulfide bond formationIntegrin-mediated cell survivalExtracellular matrixSulfhydryl oxidase 1Single-chain variantDisulfide bond formationOxidase domainTumor cell adhesionTight-binding inhibitorProtein dissectionSulfhydryl oxidase activityExtracellular matrix compositionOver-producedCell migrationCell survivalLaminin isoformsRecombinant versionCell adhesionInhibitory antibodiesCultured fibroblastsPotential therapeutic applicationsOxidase activityEnzymeLaminin
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