2024
The 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
2010
Seeing with new eyes: Finding a path to early intervention trials in Alzheimer's disease
Cedarbaum JM, Crans G, Grundman M. Seeing with new eyes: Finding a path to early intervention trials in Alzheimer's disease. The Journal Of Nutrition, Health & Aging 2010, 14: 306-309. PMID: 20306002, DOI: 10.1007/s12603-010-0069-y.Peer-Reviewed Original ResearchConceptsMild cognitive impairmentAlzheimer's diseaseOutcome measuresClinical Dementia Rating SumClinical staging algorithmsPrimary outcome measureAD clinical trialsEarly intervention trialsComprehensive outcome measuresIdentification of MCIClinical courseIntervention trialsAD patientsClinical trialsDementia pathologyClinical characterizationCognitive impairmentEarly identificationDegenerative disordersNormal cognitionStaging algorithmDiseaseBiomarker informationBiomarker dataTrials
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