2024
Non-ceruloplasmin copper and urinary copper in clinically stable Wilson disease: Alignment with recommended targets
Ott P, Sandahl T, Ala A, Cassiman D, Couchonnal-Bedoya E, Cury R, Czlonkowska A, Denk G, D’Inca R, de Assis Aquino Gondim F, Moore J, Poujois A, Twardowschy C, Weiss K, Zuin M, Kamlin C, Schilsky M. Non-ceruloplasmin copper and urinary copper in clinically stable Wilson disease: Alignment with recommended targets. JHEP Reports 2024, 6: 101115. PMID: 39139457, PMCID: PMC11321293, DOI: 10.1016/j.jhepr.2024.101115.Peer-Reviewed Original ResearchNon-ceruloplasmin-bound copperUrinary copper excretionD-penicillamine therapyRecommended target rangeWD patientsWilson's diseaseSigns of copper deficiencyD-penicillamineBiochemical signsCopper excretionTarget rangeClinically stable diseaseTreatment of WDTreatment of patientsAnalysis of liver enzymesCopper deficiencyStable diseaseMaintenance therapyScreening visitUrinary copperTreated WDHealthy controlsNormal rangeProtein speciationRecommended targets
2023
P21 Monitoring maintenance therapy with D-Penicillamine for Wilson’s Disease: lessons from screening for a randomized trial
Ala A, Yin J, Moore J, Medici V, González-Peralta R, Kamlin C, Heifetz M, Ott P, Schilsky M. P21 Monitoring maintenance therapy with D-Penicillamine for Wilson’s Disease: lessons from screening for a randomized trial. 2023, a25.2-a26. DOI: 10.1136/gutjnl-2023-basl.37.Peer-Reviewed Original Research
2015
Prospective Pilot Study of a Single Daily Dosage of Trientine for the Treatment of Wilson Disease
Ala A, Aliu E, Schilsky ML. Prospective Pilot Study of a Single Daily Dosage of Trientine for the Treatment of Wilson Disease. Digestive Diseases And Sciences 2015, 60: 1433-1439. PMID: 25605552, PMCID: PMC4427615, DOI: 10.1007/s10620-014-3495-6.Peer-Reviewed Original ResearchMeSH KeywordsAdministration, OralAdultAgedChelating AgentsDrug Administration ScheduleFemaleHepatolenticular DegenerationHumansMaleMedication AdherenceMiddle AgedPatient DropoutsPatient SatisfactionPilot ProjectsProspective StudiesSurveys and QuestionnairesTime FactorsTreatment OutcomeTrientineYoung AdultConceptsSingle daily doseWilson's diseaseDaily doseDaily treatment regimenStable Wilson's diseaseProspective pilot studyLiver synthetic functionSingle daily dosageEnd of treatmentResultsAll patientsMaintenance therapyTreatment regimenDaily dosageLifelong therapyLarge trialsZinc excretionUrine copperTreatment stoppageSide effectsTreatment efficacyTrientineTherapyPatientsPilot studyDisease
2008
Tetrathiomolybdate versus Trientine in the Initial Treatment of Neurologic Wilson's Disease
Brewer G, Askari F, Lorincz M, Carlson M, Schilsky M, Kluin K, Hedera P, Moretti P, Fink J, Tankanow R, Dick R, Sitterly J. Tetrathiomolybdate versus Trientine in the Initial Treatment of Neurologic Wilson's Disease. 2008, 153-166. DOI: 10.1017/cbo9780511666971.010.Peer-Reviewed Original ResearchTypes of patientsWilson's diseaseMaintenance therapyInitial treatmentZinc maintenance therapyDouble-blind trialOpen-label studyNeurologic Wilson diseaseWeeks of treatmentDouble-blind designAnticopper drugNeurologic worseningTM armNeurologic deteriorationIntolerant patientsMost patientsNeurologic presentationNeurologic symptomsIll patientsSuch patientsUrine studiesBlind designBlind trialPatientsLabel study
2001
Treatment of Wilson’s disease: What are the relative roles of penicillamine, trientine, and zinc supplementation?
Schilsky M. Treatment of Wilson’s disease: What are the relative roles of penicillamine, trientine, and zinc supplementation? Current Gastroenterology Reports 2001, 3: 54-59. PMID: 11177695, DOI: 10.1007/s11894-001-0041-4.Peer-Reviewed Original ResearchConceptsWilson's diseaseSymptomatic patientsTreatment of choiceInitial therapyMaintenance therapyPregnant patientsCombination therapyZinc supplementationBest therapyNeurologic diseaseChelation therapyNext treatmentMedical treatmentPatientsTherapyDiseaseTrientineFurther studiesTreatmentMore effective alternativesEffective alternativeNew optionsPenicillamineLiverSupplementation