2023
Relative Meaningfulness and Impacts of Symptoms in People with Early-Stage Parkinson’s Disease
Mammen J, Speck R, Stebbins G, Müller M, Yang P, Campbell M, Cosman J, Crawford J, Dam T, Hellsten J, Jensen-Roberts S, Kostrzebski M, Simuni T, Barowicz K, Cedarbaum J, Dorsey E, Stephenson D, Adams J. Relative Meaningfulness and Impacts of Symptoms in People with Early-Stage Parkinson’s Disease. Journal Of Parkinson's Disease 2023, 13: 619-632. PMID: 37212071, PMCID: PMC10357209, DOI: 10.3233/jpd-225068.Peer-Reviewed Original ResearchConceptsEarly Parkinson's diseaseMeaningful symptomsParkinson's diseaseEarly-stage Parkinson's diseaseMost bothersome symptomsImpact of symptomsStage Parkinson's diseaseBothersomeness of symptomsFine motor difficultiesBothersome symptomsImpact of diseasePatient's perspectiveImportant symptomNew therapiesFuture symptomsSymptom mappingSymptomsMotor difficultiesDiseaseExperiences of peopleJob functioning
2021
Quantitative digital clock drawing test as a sensitive tool to detect subtle cognitive impairments in early stage Parkinson's disease
Schejter-Margalit T, Kizony R, Shirvan J, Cedarbaum J, Bregman N, Thaler A, Giladi N, Mirelman A. Quantitative digital clock drawing test as a sensitive tool to detect subtle cognitive impairments in early stage Parkinson's disease. Parkinsonism & Related Disorders 2021, 90: 84-89. PMID: 34416663, DOI: 10.1016/j.parkreldis.2021.08.002.Peer-Reviewed Original ResearchMeSH KeywordsAdultAgedAged, 80 and overCase-Control StudiesCognitionCognitive DysfunctionFemaleGenotypeGlucosylceramidaseHumansLeucine-Rich Repeat Serine-Threonine Protein Kinase-2MaleMental Status and Dementia TestsMiddle AgedMutationNeuropsychological TestsParkinson DiseaseReproducibility of ResultsROC CurveSensitivity and SpecificityConceptsColor Trails TestSubtle cognitive declineClock Drawing TestCognitive AssessmentCognitive profileCognitive declineCognitive testsDigital clock drawing testStandardized neuropsychological testsDrawing TestDigital cognitive assessmentSubtle cognitive impairmentCurrent standardized testsClock-drawing testEarly cognitive declineMontreal Cognitive AssessmentTrails TestNeuropsychological testsSubtle impairmentsHealthy controls
2019
Randomized phase I clinical trial of anti–α‐synuclein antibody BIIB054
Brys M, Fanning L, Hung S, Ellenbogen A, Penner N, Yang M, Welch M, Koenig E, David E, Fox T, Makh S, Aldred J, Goodman I, Pepinsky B, Liu Y, Graham D, Weihofen A, Cedarbaum JM. Randomized phase I clinical trial of anti–α‐synuclein antibody BIIB054. Movement Disorders 2019, 34: 1154-1163. PMID: 31211448, PMCID: PMC6771554, DOI: 10.1002/mds.27738.Peer-Reviewed Original ResearchConceptsParkinson's disease participantsΑ-synucleinHealthy volunteersParkinson's diseaseHuman-derived monoclonal antibodiesSingle-dose cohortsMost adverse eventsFurther clinical developmentImmunotherapy targetingStudy drugAdverse eventsFavorable safetySingle doseNeuronal dysfunctionSerum ratioDisease progressionCerebrospinal fluidClinical developmentPharmacokinetic parametersPharmacokinetic profileSerum exposureLaboratory assessmentMonoclonal antibodiesDiseaseDose range
2018
FDG PET Parkinson’s disease-related pattern as a biomarker for clinical trials in early stage disease
Matthews DC, Lerman H, Lukic A, Andrews RD, Mirelman A, Wernick MN, Giladi N, Strother SC, Evans KC, Cedarbaum JM, Even-Sapir E. FDG PET Parkinson’s disease-related pattern as a biomarker for clinical trials in early stage disease. NeuroImage Clinical 2018, 20: 572-579. PMID: 30186761, PMCID: PMC6120603, DOI: 10.1016/j.nicl.2018.08.006.Peer-Reviewed Original ResearchConceptsHealthy controlsDisease-related patternsParkinson's diseaseYahr stagePD patientsClinical trialsParkinson's disease-related patternFluorodeoxyglucose positron emission tomographyGender-matched healthy controlsEarly-stage Parkinson's diseaseMild PD patientsEarly-stage diseaseFDG-PET scansMotor symptom scoresPositron emission tomographyStage diseaseMotor symptomsSymptom scoresFDG-PETDisease stagePD progressionLentiform nucleusParacentral gyrusSymptom evaluationPD subjects
2015
Item response theory analysis of the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised in the Pooled Resource Open-Access ALS Clinical Trials Database
Bacci ED, Staniewska D, Coyne KS, Boyer S, White LA, Zach N, Cedarbaum JM, Consortium T. Item response theory analysis of the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised in the Pooled Resource Open-Access ALS Clinical Trials Database. Amyotrophic Lateral Sclerosis And Frontotemporal Degeneration 2015, 17: 157-167. PMID: 26473473, DOI: 10.3109/21678421.2015.1095930.Peer-Reviewed Original ResearchConceptsAmyotrophic Lateral Sclerosis Functional Rating Scale-RevisedPooled Resource Open-Access ALS Clinical Trials DatabaseClinical trials databasesTrials databasesAmyotrophic Lateral Sclerosis Functional RatingModern test theory approachesLevel of disabilityALSFRS-R itemsMean ageDisability levelFunctional ratingGross motorDisability severitySevere disabilityPatientsConfirmatory factor analysisItem response theory analysisTreatment effectsDisabilityDomain itemsItem response categoriesResponse categoriesALSFRSInstrument's specificitySpecificity
2012
Safety, tolerability and pharmacodynamics of a skeletal muscle activator in amyotrophic lateral sclerosis
Shefner J, Cedarbaum JM, Cudkowicz ME, Maragakis N, Lee J, Jones D, Watson ML, Mahoney K, Chen M, Saikali K, Mao J, Russell AJ, Hansen RL, Malik F, Wolff AA, Team F. Safety, tolerability and pharmacodynamics of a skeletal muscle activator in amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis And Frontotemporal Degeneration 2012, 13: 430-438. PMID: 22591195, DOI: 10.3109/17482968.2012.684214.Peer-Reviewed Original ResearchConceptsAmyotrophic lateral sclerosisSingle dosesGlobal ImpressionLateral sclerosisFast skeletal muscle troponin activatorFrequent adverse eventsDose-related fashionLimb muscle strengthMaximum voluntary ventilationDose-dependent benefitMeasures of enduranceAdverse eventsPulmonary functionVoluntary ventilationGeneral fatigueTroponin activatorMuscle strengthPharmacodynamic markersHandgrip endurancePatientsRandom orderMaximal strengthDosesTolerabilityFurther studies
2010
Phase 1 Study of Aflibercept Administered Subcutaneously to Patients with Advanced Solid Tumors
Tew WP, Gordon M, Murren J, Dupont J, Pezzulli S, Aghajanian C, Sabbatini P, Mendelson D, Schwartz L, Gettinger S, Psyrri A, Cedarbaum JM, Spriggs DR. Phase 1 Study of Aflibercept Administered Subcutaneously to Patients with Advanced Solid Tumors. Clinical Cancer Research 2010, 16: 358-366. PMID: 20028764, PMCID: PMC4211604, DOI: 10.1158/1078-0432.ccr-09-2103.Peer-Reviewed Original ResearchConceptsAdvanced solid tumorsSolid tumorsDrug-related grade 3Vascular endothelial growth factor trapDose of afliberceptDose-escalation studyDose-proportional increaseInjection site reactionsPhase 1 studyManageable side effectsVascular endothelial growth factorWarrants further evaluationFavorable pharmacokinetic profileProgression of diseaseNovel antiangiogenic agentsEndothelial growth factorCommon toxicitiesStable diseasePulmonary embolismCerebral ischemiaSubcutaneous dosesSafety profileSingle doseSite reactionsSubcutaneous formulation
2005
NT-3 promotes nerve regeneration and sensory improvement in CMT1A mouse models and in patients
Sahenk Z, Nagaraja HN, McCracken BS, King WM, Freimer ML, Cedarbaum JM, Mendell JR. NT-3 promotes nerve regeneration and sensory improvement in CMT1A mouse models and in patients. Neurology 2005, 65: 681-689. PMID: 16157899, DOI: 10.1212/01.wnl.0000171978.70849.c5.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAnimalsCharcot-Marie-Tooth DiseaseDisease Models, AnimalDouble-Blind MethodFemaleHumansMaleMiceMice, Neurologic MutantsMice, NudeMice, TransgenicMiddle AgedMyelin ProteinsNerve RegenerationNeurotrophin 3Pilot ProjectsRecovery of FunctionSciatic NeuropathySural NerveTransplantation, HeterologousTreatment OutcomeConceptsNeuropathy Impairment ScoreNT-3 groupNT-3 treatmentNeurotrophin-3Placebo groupAnimal modelsNerve regenerationNude mice axonsQuantitative muscle testingSural nerve biopsyPrimary outcome measurePeripheral myelin proteinPilot clinical studyTooth type 1ANerve biopsyMuscle testingAxonal regenerationClinical studiesImpairment scoresOutcome measuresPreclinical studiesMouse modelEndpoint measuresElectrophysiologic measurementsNude mice
2003
Neurotrophin-3 Improves Functional Constipation
Parkman HP, Rao SS, Reynolds JC, Schiller LR, Wald A, Miner PB, Lembo AJ, Gordon JM, Drossman DA, Waltzman L, Stambler N, Cedarbaum JM. Neurotrophin-3 Improves Functional Constipation. The American Journal Of Gastroenterology 2003, 98: ajg2003312. PMID: 12818279, DOI: 10.1111/j.1572-0241.2003.t01-1-07477.x.Peer-Reviewed Original ResearchConceptsComplete bowel movementsNeurotrophin-3Bowel movementsColon transitFunctional constipationChronic constipationStool frequencyPlacebo-controlled phase II studyTransient injection site reactionsConstipation-related symptomsFrequent adverse eventsPhase II studyThird of patientsInjection site reactionsEnd of treatmentDose-related effectsConstipated subjectsBowel functionPrimary endpointAdverse eventsII studyImproved symptomsWeekly dosingNeurotrophic factorConstipation
2000
Recombinant human neurotrophic factors accelerate colonic transit and relieve constipation in humans
Coulie B, Szarka L, Camilleri M, Burton D, McKinzie S, Stambler N, Cedarbaum J. Recombinant human neurotrophic factors accelerate colonic transit and relieve constipation in humans. Gastroenterology 2000, 119: 41-50. PMID: 10889153, DOI: 10.1053/gast.2000.8553.Peer-Reviewed Original ResearchConceptsNeurotrophic factorStool frequencyColonic transitR-metHuBDNFHealthy subjectsBrain-derived neurotrophic factorHuman brain-derived neurotrophic factorHuman gut motilityOverall colonic transitPassage of stoolRecombinant human brain-derived neurotrophic factorGastrointestinal motor functionSmall bowel transitInjection site reactionsWeeks of treatmentNeurotrophic factor 3Neurotrophin administrationBowel transitRandomized studyGut motilityColonic emptyingSite reactionsMotor functionConstipationHealthy people
1999
A retrospective study of percutaneous endoscopic gastrostomy in ALS patients during the BDNF and CNTF trials
Kasarskis E, Scarlata D, Hill R, Fuller C, Stambler N, Cedarbaum J, in the . B. A retrospective study of percutaneous endoscopic gastrostomy in ALS patients during the BDNF and CNTF trials. Journal Of The Neurological Sciences 1999, 169: 118-125. PMID: 10540019, DOI: 10.1016/s0022-510x(99)00230-0.Peer-Reviewed Original ResearchConceptsPercutaneous endoscopic gastrostomyALS patientsEndoscopic gastrostomyClinical statusRetrospective studyVital capacityNutritional interventionNutritional supplementationRetrospective analysisPEG insertionPatientsBDNF studiesRate of declineDisease statusEarly interventionMarked reductionWeight lossGastrostomyCNTFInterventionReduction of functionSequential measurementsDaysStatusDysphagiaThe ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function
Cedarbaum J, Stambler N, Malta E, Fuller C, Hilt D, Thurmond B, Nakanishi A, . B. The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function. Journal Of The Neurological Sciences 1999, 169: 13-21. PMID: 10540002, DOI: 10.1016/s0022-510x(99)00210-5.Peer-Reviewed Original ResearchConceptsALS Functional Rating ScaleAmyotrophic lateral sclerosisFunctional Rating ScaleRating ScaleALSFRS-R scoreProgression of disabilitySickness Impact ProfileQuality of lifeVentilatory supportImpact ProfileRespiratory functionLateral sclerosisQuality of functionStrong internal consistencyAdditional assessmentInternal consistencyRating instrumentStrong determinantOrthopneaDyspneaSclerosisPatientsDysfunctionRespiratory
1992
Clinical and Pharmacokinetic Aspects of High Dose Oral Baclofen Therapy
Aisen M, Dietz M, Rossi P, Cedarbaum J, Kutt H. Clinical and Pharmacokinetic Aspects of High Dose Oral Baclofen Therapy. Journal Of Spinal Cord Medicine 1992, 15: 211-216. PMID: 1431867, DOI: 10.1080/01952307.1992.11761520.Peer-Reviewed Original ResearchConceptsHigh-dose baclofenDesk ReferenceAdequate symptomatic reliefDosage of baclofenPotential renal insufficiencyTreatment of spasticityPattern of prescriptionPeak plasma levelsPhysicians' Desk ReferenceBaclofen levelsBaclofen therapyNeurogenic bladderRenal insufficiencySymptomatic reliefBlood levelsMuscle relaxantsPlasma levelsPharmacokinetic aspectsRenal clearanceClinical practiceBaclofenPatientsPilot studyPrior reportsUseful role
1990
General assay for phosphoproteins in cerebrospinal fluid: A candidate market for paraneoplastic cerebellar degeneration
Gandy S, Grebb J, Rosen N, Albert K, Devinsky O, Blumberg H, Anderson N, Cedarbaum J, Porter R, Sedvall G, Posner J, Greengard P. General assay for phosphoproteins in cerebrospinal fluid: A candidate market for paraneoplastic cerebellar degeneration. Annals Of Neurology 1990, 28: 829-833. PMID: 2285268, DOI: 10.1002/ana.410280616.Peer-Reviewed Original ResearchConceptsProtein kinase C substrate proteinProtein kinase substratesProtein phosphorylation systemsSubstrate proteinsKinase substratePhosphorylation systemBiochemical characterizationCell typesPhosphoproteinNeuronal cellsNeuronal diseasesNeuronal enrichmentGeneral assayUnique markerPotential insightsNeuronal populationsProteinNeurological diseasesCerebellar degenerationCellsAssaysEnrichmentAssay techniquesKdMarkersThe metabolic anatomy of Parkinson's disease: Complementary [18F]fluorodeoxyglucose and [18F]fluorodopa positron emission tomographic studies
Eidelberg D, Moeller JR, Dhawan V, Sidtis JJ, Ginos JZ, Strother SC, Cedarbaum J, Greene P, Fahn S, Rottenberg DA. The metabolic anatomy of Parkinson's disease: Complementary [18F]fluorodeoxyglucose and [18F]fluorodopa positron emission tomographic studies. Movement Disorders 1990, 5: 203-213. PMID: 2117706, DOI: 10.1002/mds.870050304.Peer-Reviewed Original ResearchConceptsPositron emission tomographyPD patientsParkinson's diseaseScaled Subprofile ModelMetabolic anatomyFDOPA uptakeFDG/PETPositron emission tomographic studiesStriatal FDOPA uptakeTypical Parkinson's diseaseOverall disease severityEmission tomographic studiesGait disturbanceTwo-compartment modelBrain uptakePlasma radioactivityClinical measuresMetabolic asymmetryPET scansDisease processMotor asymmetryLeft-right differencesDisease severityEmission tomographySubprofile Model
1987
Controlled-release levodopa/carbidopa. I. Sinemet CR3 treatment of response fluctuations in Parkinson's disease.
Cedarbaum J, Breck L, Kutt H, McDowell F. Controlled-release levodopa/carbidopa. I. Sinemet CR3 treatment of response fluctuations in Parkinson's disease. Neurology 1987, 37: 233-41. PMID: 3808304, DOI: 10.1212/wnl.37.2.233.Peer-Reviewed Original ResearchConceptsPlasma levodopa levelsLevodopa levelsResponse fluctuationsParkinson's diseaseControlled-release carbidopa/levodopaCarbidopa/levodopaOpen-label trialBioavailability of levodopaStandard SinemetDisability scoresLevodopa preparationsInterdose intervalDaily dosesParkinson's patientsControlled-release formulationClinical performanceDiseaseLevodopaDay consistencyOverall benefitSinemetPatientsDosesTrialsWeeks