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
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