2021
Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment
Weinreb NJ, Camelo JS, Charrow J, McClain MR, Mistry P, Belmatoug N, investigators F. Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment. Molecular Genetics And Metabolism 2021, 132: 100-111. PMID: 33485799, DOI: 10.1016/j.ymgme.2020.12.295.Peer-Reviewed Original ResearchConceptsBone painNon-splenectomized patientsType 1 patientsBone crisesPlatelet countLiver volumeSubset analysisBody mass index (BMI) outcomesGaucher diseaseEarly treatment yearsInitial clinical improvementDifferent patient subsetsPre-treatment baselineLong-term treatmentEnzyme replacement therapyICGG Gaucher RegistryGD type 1 patientsImiglucerase treatmentAdult patientsClinical improvementSplenectomy statusGaucher RegistryPatient subsetsTreatment initiationNormal weight
2016
Molecular Stratification by Gene Expression as a Paradigm for Precision Medicine in Systemic Sclerosis
Hinchcliff M, Whitfield M. Molecular Stratification by Gene Expression as a Paradigm for Precision Medicine in Systemic Sclerosis. 2016, 657-670. DOI: 10.1007/978-3-319-31407-5_49.Peer-Reviewed Original ResearchSystemic sclerosisPatient subsetsProgressive interstitial lung diseaseDiffuse cutaneous systemic sclerosisAppropriate patient subsetsScleroderma renal crisisCutaneous systemic sclerosisInvestigator-initiated trialPulmonary arterial hypertensionSubset of patientsDifferent patient subsetsInterstitial lung diseaseDifferent pathophysiological processesMolecular pathwaysPrecision medicineGene expression subsetsT lymphocyte activationUninvolved skin samplesInflammatory subsetRenal crisisSSc complicationsArterial hypertensionMycophenolate mofetilClinical improvementOrgan involvement
2010
Development of Candidate Genomic Markers to Select Breast Cancer Patients for Dasatinib Therapy
Moulder S, Yan K, Huang F, Hess KR, Liedtke C, Lin F, Hatzis C, Hortobagyi GN, Symmans WF, Pusztai L. Development of Candidate Genomic Markers to Select Breast Cancer Patients for Dasatinib Therapy. Molecular Cancer Therapeutics 2010, 9: 1120-1127. PMID: 20423993, DOI: 10.1158/1535-7163.mct-09-1117.Peer-Reviewed Original ResearchMeSH KeywordsAntineoplastic AgentsBiomarkers, PharmacologicalBiomarkers, TumorBreast NeoplasmsCarcinomaCell Line, TumorDasatinibDrug Resistance, NeoplasmFemaleGene Expression ProfilingGene Expression Regulation, NeoplasticGenetic Association StudiesGenome, HumanHumansMatched-Pair AnalysisOligonucleotide Array Sequence AnalysisPatient SelectionPrognosisPyrimidinesThiazolesConceptsClinical trialsCell linesPhase I/II trialIndependent breast cancer cell linesEarly phase clinical trialsDasatinib-resistant cellsPrimary breast cancerBreast cancer patientsDasatinib-resistant cell linesDifferent patient subsetsBreast cancer cell linesGenomic predictorsCancer cell linesDasatinib therapyDifferent potential predictorsII trialPatient subsetsPatient selectionCancer patientsBreast cancerDasatinib sensitivityMammary epithelial cellsDasatinib responseActivity indexPatient samples
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