2024
Validating new symptom emergence as a patient-centric outcome measure for PD clinical trials
Zou H, Stebbins G, Simuni T, Luo S, Cedarbaum J. Validating new symptom emergence as a patient-centric outcome measure for PD clinical trials. Parkinsonism & Related Disorders 2024, 128: 107118. PMID: 39353265, DOI: 10.1016/j.parkreldis.2024.107118.Peer-Reviewed Original ResearchPD clinical trialsClinical trialsEmergent symptomsPhase 3 clinical trialsPhase 3 studySlowing of disease progressionOutcome measuresParkinson's diseaseDe novo Parkinson's diseaseMDS-UPDRSItem-level dataFrequency of ESSymptomatic medicationsFrequent administrationMonths of observationEfficacy assessmentDisease progressionSymptom emergenceRating ScaleItem responsesIb and IIUrate elevationTrialsThe ALSFRS-R Summit: a global call to action on the use of the ALSFRS-R in ALS clinical trials
Genge A, Cedarbaum J, Shefner J, Chio A, Al-Chalabi A, Van Damme P, McDermott C, Glass J, Berry J, van Eijk R, Fournier C, Grosskreutz J, Andrews J, Bertone V, Bunte T, Couillard M, Cummings C, Kittle G, Polzer J, Salmon K, Straub C, van den Berg L. The ALSFRS-R Summit: a global call to action on the use of the ALSFRS-R in ALS clinical trials. Amyotrophic Lateral Sclerosis And Frontotemporal Degeneration 2024, 25: 382-387. PMID: 38396337, DOI: 10.1080/21678421.2024.2320880.Peer-Reviewed Original ResearchAmyotrophic Lateral Sclerosis Functional Rating ScaleALSFRS-RClinical trialsStatistical analysis planMonitor functional changesRating ScaleOutcome measuresFunctional Rating ScalePrimary outcome measureClinical trial designALS clinical trialsFunctional changesTrial designTrialsClinical trialistsScaleALS trials
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 levelsTracking Emergence of New Motor and Non-Motor Symptoms Using the MDS-UPDRS: A Novel Outcome Measure for Early Parkinson’s Disease?
Tosin M, Simuni T, Stebbins G, Cedarbaum J. Tracking Emergence of New Motor and Non-Motor Symptoms Using the MDS-UPDRS: A Novel Outcome Measure for Early Parkinson’s Disease? Journal Of Parkinson's Disease 2022, 12: 1345-1351. PMID: 35466955, PMCID: PMC9198734, DOI: 10.3233/jpd-223170.Peer-Reviewed Original ResearchConceptsEmergent symptomsDisease progressionOutcome measuresParkinson's disease clinical trialsNon-motor symptomsEarly Parkinson's diseaseDaily Living ScalePatient-reported experiencesClinical rating scalesNovel outcome measuresSymptomatic treatmentPD progressionClinical trialsMedian numberDaily livingLiving ScaleParkinson's diseaseSummary scoresMDS-UPDRSUseful markerRating ScaleSTX groupProgressionStxSymptoms
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 scoresPresentationMutationsSymptoms
1999
The 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
1991
Effect of MK-458 (HPMC) in Parkinson's Disease Previously Untreated With Dopaminergic Drugs. A Double-Blind, Placebo-Controlled Multicenter Study.
Koller WC, Block GA, Ahlskog JE, Ahrens S, Cedarbaum JM, Cyhan G, Goetz CG, LeWitt PA, Liss C, McLean L, Moses H, Muenter M, Nausieda P, Sanchez-Ramos J, Reich S, Singer C, Stellar S, Weiner W. Effect of MK-458 (HPMC) in Parkinson's Disease Previously Untreated With Dopaminergic Drugs. A Double-Blind, Placebo-Controlled Multicenter Study. Clinical Neuropharmacology 1991, 14: 322. PMID: 1913699, DOI: 10.1097/00002826-199108000-00004.Peer-Reviewed Original ResearchConceptsMK-458Early Parkinson's diseaseParkinson's diseaseDopaminergic drugsDisability Rating ScoreDopamine receptor agonismPlacebo-controlled evaluationPatient global assessmentClinical rating scalesParkinsonian symptomsAntiparkinsonian efficacyMulticenter studyAdverse reactionsIdeal treatmentReceptor agonistReceptor agonismSustained release formulationPlaceboGlobal assessmentDiseaseRating ScaleSignificant decreaseDrugsPatientsRating scores