2022
Longitudinal clinical and biomarker characteristics of non-manifesting LRRK2 G2019S carriers in the PPMI cohort
Siderowf A, Seibyl J, Coffey C, Tosun-Turgut D, Shaw L, Trojanowski J, Singleton A, Kieburtz K, Toga A, Mollenhauer B, Galasko D, Poewe W, Foroud T, Poston K, Bressman S, Reimer A, Arnedo V, Clark A, Frasier M, Kopil C, Chowdhury S, Casaceli C, Dorsey R, Wilson R, Mahes S, Seibyl J, Salerno C, Ahrens M, Brumm M, Cho H, Fedler J, LaFontant D, Kurth R, Crawford K, Casalin P, Malferrari G, Weisz M, Orr-Urtreger A, Trojanowski J, Shaw L, Montine T, Baglieri C, Christini A, Russell D, Dahodwala N, Giladi N, Factor S, Hogarth P, Standaert D, Hauser R, Jankovic J, Saint-Hilaire M, Richard I, Shprecher D, Fernandez H, Brockmann K, Rosenthal L, Barone P, Espayc A, Rowe D, Marder K, Santiago A, Bressman S, Hu S, Isaacson S, Corvol J, Martinez J, Tolosa E, Tai Y, Politis M, Smejdir D, Rees L, Williams K, Kausar F, Williams K, Richardson W, Willeke D, Peacock S, Sommerfeld B, Freed A, Wakeman K, Blair C, Guthrie S, Harrell L, Hunter C, Thomas C, James R, Zimmerman G, Brown V, Mule J, Hilt E, Ribb K, Ainscough S, Wethington M, Ranola M, Santana H, Moreno J, Raymond D, Speketer K, Carvajal L, Carvalo S, Croitoru I, Garrido A, Payne L, Viswanth V, Severt L, Facheris M, Soares H, Mintun M, Cedarbaum J, Taylor P, Biglan K, Vandenbroucke E, Sheikh Z, Bingol B, Fischer T, Sardi P, Forrat R, Reith A, Egebjerg J, Hillert G, Saba B, Min C, Umek R, Mather J, De Santi S, Post A, Boess F, Taylor K, Grachev I, Avbersek A, Muglia P, Merchant K, Tauscher J. Longitudinal clinical and biomarker characteristics of non-manifesting LRRK2 G2019S carriers in the PPMI cohort. Npj Parkinson's Disease 2022, 8: 140. PMID: 36273008, PMCID: PMC9588016, DOI: 10.1038/s41531-022-00404-w.Peer-Reviewed Original ResearchNon-manifesting carriersParkinson's Progression Markers InitiativeHealthy controlsDAT deficitProgression Markers InitiativeLongitudinal changesBiofluid biomarkersCerebrospinal fluid AbetaNon-motor scalesDopamine transporter imagingPrevention clinical trialsNeurofilament light chainLRRK2 G2019S carriersClinical featuresClinical outcomesPhospho-tauTransporter imagingPPMI cohortTotal tauDAT bindingMean ageSerum biomarkersClinical trialsClinical measuresComprehensive motor
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 ResearchConceptsAmyotrophic 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