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 ResearchMeSH KeywordsAmyotrophic Lateral SclerosisCross-Sectional StudiesHumansInternetLanguageSurveys and QuestionnairesConceptsAmyotrophic Lateral Sclerosis Functional Rating ScaleFunctional Rating ScaleOutcome measuresRating ScaleWeb-based cross-sectional surveyAbility of patientsCross-sectional surveyALS studiesWeb-based surveyALSFRSPatient communityPerspectives of peopleCaregiversPatientsALSItem questionsLiteracy levels
2021
Systematic Assessment of 10 Biomarker Candidates Focusing on α‐Synuclein‐Related Disorders
Schulz I, Kruse N, Gera R, Kremer T, Cedarbaum J, Barbour R, Zago W, Schade S, Otte B, Bartl M, Hutten S, Trenkwalder C, Mollenhauer B. Systematic Assessment of 10 Biomarker Candidates Focusing on α‐Synuclein‐Related Disorders. Movement Disorders 2021, 36: 2874-2887. PMID: 34363416, DOI: 10.1002/mds.28738.Peer-Reviewed Original ResearchMeSH KeywordsAlpha-SynucleinAmyotrophic Lateral SclerosisBiomarkersFrontotemporal DementiaHumansMultiple System AtrophyTau ProteinsConceptsGlial fibrillary acidic proteinCerebrospinal fluidChitinase-3-like protein 1Calcium-binding protein BUbiquitin C-terminal hydrolase L1Myeloid cells 2Neurofilament light chainObjective diagnostic biomarkersFibrillary acidic proteinNeurofilament heavy chainDiagnostic biomarker candidatesDiagnostic biomarkersClinical diagnosisTau proteinPhosphorylated aSynNeurodegenerative disordersAcidic proteinCells 2Α-synucleinBiomarker candidatesProtein 1Candidate markersAvailable kitsDisordersLight chain
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 ResearchMeSH KeywordsAdolescentAdultAgedAged, 80 and overAmyotrophic Lateral SclerosisClinical Trials as TopicDatabases, FactualFemaleHealth ResourcesHumansMaleMiddle AgedModels, StatisticalYoung AdultConceptsAmyotrophic 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 ResearchMeSH KeywordsAdultAgedAmyotrophic Lateral SclerosisFemaleHand StrengthHumansImidazolesMaleMiddle AgedMuscle, SkeletalNeuroprotective AgentsPyrazinesRiluzoleTreatment OutcomeConceptsAmyotrophic 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
2004
Survival
Cedarbaum J. Survival. Amyotrophic Lateral Sclerosis And Frontotemporal Degeneration 2004, 5: 79-83. PMID: 15512880, DOI: 10.1080/17434470410020021.Peer-Reviewed Original ResearchAmyotrophic Lateral SclerosisClinical Trials as TopicEndpoint DeterminationHumansMeta-Analysis as TopicTime Factors
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
1998
Prognostic indicators of survival in ALS
Stambler N, Charatan M, Cedarbaum J. Prognostic indicators of survival in ALS. Neurology 1998, 50: 66-72. PMID: 9443459, DOI: 10.1212/wnl.50.1.66.Peer-Reviewed Original ResearchConceptsStudy entrySerum chlorideMuscle strength lossBaseline demographic characteristicsLength of survivalRisk of mortalityClinical laboratory testsGreater weight lossCiliary neurotrophic factorRecombinant human ciliary neurotrophic factorHuman ciliary neurotrophic factorPlacebo groupPulmonary functionSymptom onsetPrognostic factorsMulticenter studyShorter survivalVital capacityNeurotrophic factorPrognostic indicatorRespiratory acidosisSurvival timeProbability of survivalDisease severityWeight loss
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