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 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 ResearchA 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 Research
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
Intrathecal Ciliary Neurotrophic Factor Delivery for Treatment of Amyotrophic Lateral Sclerosis (Phase I Trial)
Penn R, Kroin J, York M, Cedarbaum J. Intrathecal Ciliary Neurotrophic Factor Delivery for Treatment of Amyotrophic Lateral Sclerosis (Phase I Trial). Neurosurgery 1997, 40: 94-100. DOI: 10.1227/00006123-199701000-00021.Peer-Reviewed Original Research