2022
A qualitative evaluation of the revised amyotrophic lateral sclerosis functional rating scale (ALSFRS-R) by the patient community: a web-based cross-sectional survey
Boyce D, Robinson M, Cedarbaum J, Shank L, McDermott C, van Eijk R. A qualitative evaluation of the revised amyotrophic lateral sclerosis functional rating scale (ALSFRS-R) by the patient community: a web-based cross-sectional survey. Amyotrophic Lateral Sclerosis And Frontotemporal Degeneration 2022, 24: 272-280. PMID: 36330850, DOI: 10.1080/21678421.2022.2140592.Peer-Reviewed Original ResearchConceptsAmyotrophic Lateral Sclerosis Functional Rating ScaleFunctional Rating ScaleOutcome measuresRating ScaleWeb-based cross-sectional surveyAbility of patientsCross-sectional surveyALS studiesWeb-based surveyALSFRSPatient communityPerspectives of peopleCaregiversPatientsALSItem questionsLiteracy levelsNeuromelanin and T2*-MRI for the assessment of genetically at-risk, prodromal, and symptomatic Parkinson’s disease
Ben Bashat D, Thaler A, Lerman Shacham H, Even-Sapir E, Hutchison M, Evans K, Orr-Urterger A, Cedarbaum J, Droby A, Giladi N, Mirelman A, Artzi M. Neuromelanin and T2*-MRI for the assessment of genetically at-risk, prodromal, and symptomatic Parkinson’s disease. Npj Parkinson's Disease 2022, 8: 139. PMID: 36271084, PMCID: PMC9586960, DOI: 10.1038/s41531-022-00405-9.Peer-Reviewed Original ResearchParkinson's diseaseGenotype-related differencesDAT-SPECTRadiomic featuresSymptomatic Parkinson's diseaseSignificant correlationProdromal phaseBrain regionsNeuromelanin MRIIron accumulationDiseaseMRIAssessment of individualsImaging valuesRadiomics analysisSignificant differencesPatientsNeuromelaninRiskAgeScoresT2Ratio scoresGroupLR scoresApplication of longitudinal item response theory models to modeling Parkinson’s disease progression
Zou H, Aggarwal V, Stebbins G, Müller M, Cedarbaum J, Pedata A, Stephenson D, Simuni T, Luo S. Application of longitudinal item response theory models to modeling Parkinson’s disease progression. CPT Pharmacometrics & Systems Pharmacology 2022, 11: 1382-1392. PMID: 35895005, PMCID: PMC9574723, DOI: 10.1002/psp4.12853.Peer-Reviewed Original ResearchConceptsEarly Parkinson's diseaseParkinson's diseaseDisease progressionProgression rateUnified Parkinson's Disease Rating Scale part 2Stage 1Yahr stage 1Higher baseline severityCommon clinical outcomeLongitudinal disease progressionSlow progression rateParkinson's disease progressionMovement Disorder SocietyStage 2Clinical outcomesMotor signsBaseline severitySlow progressionSum scoreTotal scoreLongitudinal item response theory modelProgressionSeverityPatientsDisease
2021
Longitudinal Changes in Neuromelanin MRI Signal in Parkinson's Disease: A Progression Marker
Gaurav R, Yahia‐Cherif L, Pyatigorskaya N, Mangone G, Biondetti E, Valabrègue R, Ewenczyk C, Hutchison R, Cedarbaum J, Corvol J, Vidailhet M, Lehéricy S. Longitudinal Changes in Neuromelanin MRI Signal in Parkinson's Disease: A Progression Marker. Movement Disorders 2021, 36: 1592-1602. PMID: 33751655, PMCID: PMC8359265, DOI: 10.1002/mds.28531.Peer-Reviewed Original ResearchConceptsTotal intracranial volumePD patientsHealthy volunteersParkinson's diseaseIntracranial volumeObservational case-control studySubstantia nigra pars compactaDirect noninvasive assessmentDisease-modifying treatmentsEarly PD patientsFemale PD patientsTherapeutic drug trialsCase-control studyNeuromelanin-sensitive MRI techniquesNeuromelanin-sensitive imagingMale patientsFemale patientsPars compactaCohort IICohort IDrug trialsPatientsImaging biomarkersNoninvasive assessmentDisease severity
2020
A Possible Modifying Effect of the G2019S Mutation in the LRRK2 Gene on GBA Parkinson's Disease
Omer N, Giladi N, Gurevich T, Bar‐Shira A, Gana‐Weisz M, Goldstein O, Kestenbaum M, Cedarbaum JM, Orr‐Urtreger A, Mirelman A, Thaler A. A Possible Modifying Effect of the G2019S Mutation in the LRRK2 Gene on GBA Parkinson's Disease. Movement Disorders 2020, 35: 1249-1253. PMID: 32353202, DOI: 10.1002/mds.28066.Peer-Reviewed Original ResearchConceptsGBA-PDLRRK2-PDIdiopathic Parkinson's diseaseDisease Rating ScaleParkinson's diseaseTotal Unified Parkinson's Disease Rating ScaleUnified Parkinson's Disease Rating ScaleParkinson's Disease Rating ScaleG2019S LRRK2 mutationBetter olfactionClinical symptomsPatientsGBA genePhenotypic presentationRating ScalePerformance-based measuresPhenotypic expressionDiseaseLower scoresPresentationMutationsSymptomsTossing and Turning in Bed: Nocturnal Movements in Parkinson's Disease
Mirelman A, Hillel I, Rochester L, Del Din S, Bloem BR, Avanzino L, Nieuwboer A, Maidan I, Herman T, Thaler A, Gurevich T, Kestenbaum M, Orr‐Urtreger A, Brys M, Cedarbaum JM, Giladi N, Hausdorff JM. Tossing and Turning in Bed: Nocturnal Movements in Parkinson's Disease. Movement Disorders 2020, 35: 959-968. PMID: 32080891, DOI: 10.1002/mds.28006.Peer-Reviewed Original ResearchConceptsAdvanced Parkinson's diseaseParkinson's diseaseNocturnal hypokinesiaDopaminergic medicationSleep disturbancesDisease severityDiverse disease severitiesPD motor severityUpright periodsDopaminergic treatmentNonmotor symptomsUpright timeMotor severityHealthy controlsSleep interruptionDisease spectrumPatientsSeverityMedicationsHypokinesiaDiseaseNocturnal movementsDegree of rotationTri-axial accelerometerDysautonomia
2019
Revisiting the non-Gaucher-GBA-E326K carrier state: Is it sufficient to increase Parkinson's disease risk?
Goldstein O, Gana-Weisz M, Cohen-Avinoam D, Shiner T, Thaler A, Cedarbaum JM, John S, Lalioti M, Gurevich T, Bar-Shira A, Mirelman A, Giladi N, Orr-Urtreger A. Revisiting the non-Gaucher-GBA-E326K carrier state: Is it sufficient to increase Parkinson's disease risk? Molecular Genetics And Metabolism 2019, 128: 470-475. PMID: 31662221, DOI: 10.1016/j.ymgme.2019.10.001.Peer-Reviewed Original ResearchConceptsPD patientsE326KGBA variantsLRRK2 G2019SDisease riskDisease worldElderly healthy individualsAdditive effectParkinson's disease riskK variantAshkenazi PD patientsCommon genetic risk factorGenetic risk factorsRisk factorsCarrier patientsGBA mutationsPD riskHealthy individualsPatientsCarrier stateGaucher diseaseEarlier AAOAshkenazi originAshkenazi controlsStatistical significance
2018
Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Study of Anti-Alpha-Synuclein Antibody BIIB054 in Patients with Parkinson’s Disease (S26.001)
Brys M, Ellenbogen A, Fanning L, Penner N, Yang M, Welch M, Koenig E, David E, Fox T, Makh S, Aldred J, Goodman I, Graham D, Weihofen A, Cedarbaum J. Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Study of Anti-Alpha-Synuclein Antibody BIIB054 in Patients with Parkinson’s Disease (S26.001). Neurology 2018, 90 DOI: 10.1212/wnl.90.15_supplement.s26.001.Peer-Reviewed Original Research
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 studiesA Study To Evaluate Safety, and Tolerability of Repeated Doses of CK-2017357 (CK-357) in Patients with Amyotrophic Lateral Sclerosis (S25.005)
Shefner J, Barohn R, Boylan K, Bradshaw D, Brooks B, Heiman-Patterson T, Katz J, Maragakis N, Mitsumoto H, Pestronk A, Simmons Z, Wolff A, Lee J, Masonek J, Jones D, Meng L, Cedarbaum J. A Study To Evaluate Safety, and Tolerability of Repeated Doses of CK-2017357 (CK-357) in Patients with Amyotrophic Lateral Sclerosis (S25.005). Neurology 2012, 78: s25.005-s25.005. DOI: 10.1212/wnl.78.1_meetingabstracts.s25.005.Peer-Reviewed Original ResearchA Study To Evaluate Safety and Tolerability of CK-2017357 (CK-357) in Patients with Amyotrophic Lateral Sclerosis Using a Twice-Daily, Dose-Titration Regimen (P04.155)
Shefner J, Andrews J, Bedlack R, Berry J, Goslin K, Jackson C, Kissel J, Lange D, Licht J, Mozaffar T, Pestronk A, Rosenfeld J, Wolff A, Lee J, Masonek J, Jones D, Meng L, Cedarbaum J. A Study To Evaluate Safety and Tolerability of CK-2017357 (CK-357) in Patients with Amyotrophic Lateral Sclerosis Using a Twice-Daily, Dose-Titration Regimen (P04.155). Neurology 2012, 78: p04.155-p04.155. DOI: 10.1212/wnl.78.1_meetingabstracts.p04.155.Peer-Reviewed Original Research
2011
P2‐509: Population pharmacokinetic analysis of plasma, cerebrospinal fluid and brain ELND005 in patients with mild to moderate Alzheimer's disease
Wagg J, Green M, Yan L, Liang E, Cedarbaum J, Abushakra S. P2‐509: Population pharmacokinetic analysis of plasma, cerebrospinal fluid and brain ELND005 in patients with mild to moderate Alzheimer's disease. Alzheimer's & Dementia 2011, 7: s475-s475. DOI: 10.1016/j.jalz.2011.05.1380.Peer-Reviewed Original Research
2009
An exploratory study of the safety, tolerability and bioactivity of a single intravitreal injection of vascular endothelial growth factor Trap-Eye in patients with diabetic macular oedema
V D, Nguyen QD, Shah SM, Browning DJ, Haller JA, Chu K, Yang K, Cedarbaum JM, Vitti RL, Ingerman A, Campochiaro PA. An exploratory study of the safety, tolerability and bioactivity of a single intravitreal injection of vascular endothelial growth factor Trap-Eye in patients with diabetic macular oedema. British Journal Of Ophthalmology 2009, 93: 144. PMID: 19174400, DOI: 10.1136/bjo.2008.138271.Peer-Reviewed Original ResearchConceptsVEGF Trap-EyeDiabetic macular edemaSingle intravitreal injectionVascular endothelial growth factor (VEGF) Trap-EyeExcess foveal thicknessFoveal thicknessIntravitreal injectionOptical coherence tomographyMacular edemaUnrelated serious adverse eventsMedian baseline BCVASerious adverse eventsBaseline BCVAETDRS lettersAdverse eventsRetinal thicknessVisual acuityOcular toxicityOutcome measuresBCVAMedian improvementPatientsCoherence tomographyAdditional studiesObservation period
2006
Dynamic and Quantitative Analysis of Choroidal Neovascularization by Fluorescein Angiography
Shah SM, Tatlipinar S, Quinlan E, Sung JU, Tabandeh H, Nguyen QD, Fahmy AS, Zimmer-Galler I, Symons RC, Cedarbaum JM, Campochiaro PA. Dynamic and Quantitative Analysis of Choroidal Neovascularization by Fluorescein Angiography. Investigative Ophthalmology & Visual Science 2006, 47: 5460-5468. PMID: 17122137, DOI: 10.1167/iovs.06-0012.Peer-Reviewed Original ResearchConceptsFluorescein angiographyFluorescein angiogramsClinical trialsVEGF TrapVascular endothelial growth factor trapPlacebo-treated patientsPhotodynamic therapyChoroidal neovascularization lesion sizeAreas of hyperfluorescenceMacular volumeChoroidal neovascularizationRetrospective gradingOutcome measuresGroup 2Group 1Masked observersPatientsFluorescence areaLesion sizeAngiogramsClinical settingDye injectionInitial assessmentAUCTrials
2005
Safety and pharmacokinetics of intravenous VEGF Trap in a phase I clinical trial of patients with advanced solid tumors
Dupont J, Rothenberg M, Spriggs D, Cedarbaum J, Furfine E, Cohen D, Dancy I, Lee H, Cooper W, Lockhart A. Safety and pharmacokinetics of intravenous VEGF Trap in a phase I clinical trial of patients with advanced solid tumors. Journal Of Clinical Oncology 2005, 23: 3029-3029. DOI: 10.1200/jco.2005.23.16_suppl.3029.Peer-Reviewed Original Research
2001
Disease status and use of ventilatory support by ALS patients
Cedarbaum J, Stambler N. Disease status and use of ventilatory support by ALS patients. Amyotrophic Lateral Sclerosis And Frontotemporal Degeneration 2001, 2: 19-22. PMID: 11465928, DOI: 10.1080/146608201300079373.Peer-Reviewed Original ResearchConceptsMechanical ventilationPlacebo patientsUse of MVRespiratory adverse eventsUse of BiPAPHealth resource utilizationPhase III trialsBiPAP useR-metHuBDNFProspective databaseSymptom durationVentilatory supportAdverse eventsIII trialsALS patientsMean baselinePrescribing practicesVital capacityBehavior of physiciansBiPAPALSFRS scorePatientsMortality rateDisease statusTrials
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
1997
Performance of the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) in multicenter clinical trials
Cedarbaum J, Stambler N. Performance of the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) in multicenter clinical trials. Journal Of The Neurological Sciences 1997, 152: s1-s9. PMID: 9419047, DOI: 10.1016/s0022-510x(97)00237-2.Peer-Reviewed Original ResearchConceptsAmyotrophic Lateral Sclerosis Functional Rating ScaleFunctional Rating ScaleMulticenter clinical trialFunctional statusRating ScaleClinical trialsPatients' functional statusClinical Global ImpressionMuscle strength testingArea of feedingPlacebo patientsTherapeutic trialsTest-retest reliabilityGlobal ImpressionPatient levelTreatment studiesChange ScaleTrialsPatientsMaximum scoreIndependent assessmentStrength testingAmbulation