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 motorApplication of longitudinal item response theory models to modeling Parkinson’s disease progression
Zou H, Aggarwal V, Stebbins G, Müller M, Cedarbaum J, Pedata A, Stephenson D, Simuni T, Luo S. Application of longitudinal item response theory models to modeling Parkinson’s disease progression. CPT Pharmacometrics & Systems Pharmacology 2022, 11: 1382-1392. PMID: 35895005, PMCID: PMC9574723, DOI: 10.1002/psp4.12853.Peer-Reviewed Original ResearchConceptsEarly Parkinson's diseaseParkinson's diseaseDisease progressionProgression rateUnified Parkinson's Disease Rating Scale part 2Stage 1Yahr stage 1Higher baseline severityCommon clinical outcomeLongitudinal disease progressionSlow progression rateParkinson's disease progressionMovement Disorder SocietyStage 2Clinical outcomesMotor signsBaseline severitySlow progressionSum scoreTotal scoreLongitudinal item response theory modelProgressionSeverityPatientsDisease
2019
A Proposed Roadmap for Parkinson’s Disease Proof of Concept Clinical Trials Investigating Compounds Targeting Alpha-Synuclein
Merchant KM, Cedarbaum JM, Brundin P, Dave KD, Eberling J, Espay AJ, Hutten SJ, Javidnia M, Luthman J, Maetzler W, Menalled L, Reimer AN, Stoessl AJ, Weiner DM, . A Proposed Roadmap for Parkinson’s Disease Proof of Concept Clinical Trials Investigating Compounds Targeting Alpha-Synuclein. Journal Of Parkinson's Disease 2019, Preprint: 1-31. PMID: 30400107, PMCID: PMC6398545, DOI: 10.3233/jpd-181471.Peer-Reviewed Original ResearchConceptsParkinson's diseaseProgression of PDDisease-modifying therapiesConcept clinical trialMichael J. Fox FoundationDisease-modifying therapeuticsLewy pathologyClinical outcomesClinical trialsTargeting therapyAnimal modelsClinical proofAlpha-synucleinBiomarker toolkitΑ-synClinical researchInvestigational moleculesTherapyPD researchTranslational frameworkParkinson's ResearchDiseaseBiomarkersConcept studyMeaningful strides