2024
Development of a General Composite Scale (GENCOMS) for Progressive Neurodegenerative Diseases and Implications for the Assessment of Disease-Modifying Therapies
Dickson S, Mallinckrodt C, Rogula B, Powell L, Potashman M, Coric V, L’Italien G, Hendrix S. Development of a General Composite Scale (GENCOMS) for Progressive Neurodegenerative Diseases and Implications for the Assessment of Disease-Modifying Therapies. Neurology And Therapy 2024, 13: 1627-1639. PMID: 39287752, PMCID: PMC11541966, DOI: 10.1007/s40120-024-00661-2.Peer-Reviewed Original ResearchComposite scaleDisease-modifying therapiesItem-level dataDisease progressionDetect clinically meaningful effectsPlacebo arm dataValidity scalesMeasure disease progressionTreatment effectsClinical declineDMT effectClinically meaningful effectChange scoresAssess treatment effectsDetect disease progressionTreatment outcomesSlow disease progressionMeaningful effectSensitive to disease progressionNeurodegenerative disease progressionItemsResponse outcomesPatient populationStandard itemsNatural history dataTroriluzole rescues glutamatergic deficits, amyloid and tau pathology, and synaptic and memory impairments in 3xTg‐AD mice
Pfitzer J, Pinky P, Perman S, Redmon E, Cmelak L, Suppiramaniam V, Coric V, Qureshi I, Gramlich M, Reed M. Troriluzole rescues glutamatergic deficits, amyloid and tau pathology, and synaptic and memory impairments in 3xTg‐AD mice. Journal Of Neurochemistry 2024 PMID: 39214859, DOI: 10.1111/jnc.16215.Peer-Reviewed Original ResearchTau mouse modelsRestore cognitive deficitsAlzheimer's diseaseSynaptic vesiclesSV pool sizeMouse model of ADCognitive deficitsTau-related pathologyGlutamate releasePool sizeModel of ADHippocampal culturesGlutamate-modulating drugsLoss of glutamatergic synapsesBeta-amyloidGlutamate modulator riluzoleTau pathologyPresynaptic vesicular glutamate transporterVesicular glutamate transportersAD pathologyAccumulation of glutamateMouse modelTau modelStages of ADPresynaptic dysfunctionVideo-Based Kinematic Analysis of Movement Quality in a Phase 3 Clinical Trial of Troriluzole in Adults with Spinocerebellar Ataxia: A Post Hoc Analysis
L’Italien G, Oikonomou E, Khera R, Potashman M, Beiner M, Maclaine G, Schmahmann J, Perlman S, Coric V. Video-Based Kinematic Analysis of Movement Quality in a Phase 3 Clinical Trial of Troriluzole in Adults with Spinocerebellar Ataxia: A Post Hoc Analysis. Neurology And Therapy 2024, 13: 1287-1301. PMID: 38814532, PMCID: PMC11263303, DOI: 10.1007/s40120-024-00625-6.Peer-Reviewed Original ResearchMovement qualityVideo-based kinematic analysisGait stability measuresTandem walking taskRating of AtaxiaGait qualityGait assessmentGait parametersTandem walkingWalking assessmentsWalking taskSpinocerebellar ataxiaVideo-based assessmentClinical expertiseNatural walkingRandomized participantsGaitHigh riskPost hoc analysisStandardized scalesAdultsWalkingParticipantsHoc analysisPhase 3Re-weighting MDS-UPDRS Part II Items for Optimal Sensitivity to Parkinson’s Disease Progression Using Parkinson’s Progression Markers Initiative Natural History Data (P11-3.012)
Dickson S, Rogula B, Dubow J, Kozauer N, Powell L, Potashman M, Crimin K, O’Keefe P, Korol E, Crabtree M, Nagase F, Coric V, Hendrix S, L’Italien G. Re-weighting MDS-UPDRS Part II Items for Optimal Sensitivity to Parkinson’s Disease Progression Using Parkinson’s Progression Markers Initiative Natural History Data (P11-3.012). Neurology 2024, 102 DOI: 10.1212/wnl.0000000000204570.Peer-Reviewed Original ResearchFunctional Impairments in Patients with KCNQ2-DEE: Associations Among Key Clinical Features (P1-1.007)
Berg A, Lerner J, Millichap J, L’Italien G, Coric V, Potashman M. Functional Impairments in Patients with KCNQ2-DEE: Associations Among Key Clinical Features (P1-1.007). Neurology 2024, 102 DOI: 10.1212/wnl.0000000000204827.Peer-Reviewed Original ResearchPsychometric Validation of the Modified-functional Scale for the Assessment and Rating of Ataxia (P7-3.018)
Potashman M, Popoff E, Powell L, Beiner M, Mackenzie A, Coric V, Schmahmann J, L’Italien G. Psychometric Validation of the Modified-functional Scale for the Assessment and Rating of Ataxia (P7-3.018). Neurology 2024, 102 DOI: 10.1212/wnl.0000000000204549.Peer-Reviewed Original ResearchRe-weighting MDS-UPDRS Motor Items for Optimal Sensitivity to Parkinson’s Disease Progression in Untreated Patients Using Parkinson’s Progression Markers Initiative Data (S2.004)
L’Italien G, Dickson S, Rogula B, Dubow J, Kozauer N, Powell L, Potashman M, Crimin K, O’Keefe P, Korol E, Crabtree M, Nagase F, Coric V, Hendrix S. Re-weighting MDS-UPDRS Motor Items for Optimal Sensitivity to Parkinson’s Disease Progression in Untreated Patients Using Parkinson’s Progression Markers Initiative Data (S2.004). Neurology 2024, 102 DOI: 10.1212/wnl.0000000000204569.Peer-Reviewed Original ResearchNext Generation Prodrug Troriluzole: Increased Bioavailability of Riluzole with No Food Effect in Healthy Subjects (S5.006)
Sevinsky H, Rozakis R, Nepomuceno T, Malatesta J, Awsare B, Ashbrenner E, Kaplita S, Gentile K, Hussey E, Qureshi I, Coric V, Bertz R. Next Generation Prodrug Troriluzole: Increased Bioavailability of Riluzole with No Food Effect in Healthy Subjects (S5.006). Neurology 2024, 102 DOI: 10.1212/wnl.0000000000205022.Peer-Reviewed Original ResearchNo Clinically Relevant Effects of Hepatic Impairment on the Pharmacokinetics of a Next Generation Prodrug Troriluzole (P4-4.014)
Bertz R, Rozakis R, Donohue M, Sevinsky H, Awsare B, Kaplita S, Hussey E, Qureshi I, Coric V. No Clinically Relevant Effects of Hepatic Impairment on the Pharmacokinetics of a Next Generation Prodrug Troriluzole (P4-4.014). Neurology 2024, 102 DOI: 10.1212/wnl.0000000000206134.Peer-Reviewed Original ResearchTroriluzole Exhibits Favorable Hepatic Safety Profile Across a Diverse Range of Disorders (P4-4.013)
Qureshi I, Beiner M, Bertz R, Kaplita S, Yang R, Munivar A, Stock D, Wirtz V, Coric V. Troriluzole Exhibits Favorable Hepatic Safety Profile Across a Diverse Range of Disorders (P4-4.013). Neurology 2024, 102 DOI: 10.1212/wnl.0000000000206182.Peer-Reviewed Original ResearchDeterminants of Health-related Quality of Life of Patients with Focal Epilepsy: A Systematic Literature Review (P9-1.016)
Marcin C, Engelhart P, Lerner J, Dill D, L’Italien G, Coric V, Matsumoto J, Potashman M. Determinants of Health-related Quality of Life of Patients with Focal Epilepsy: A Systematic Literature Review (P9-1.016). Neurology 2024, 102 DOI: 10.1212/wnl.0000000000204828.Peer-Reviewed Original ResearchMatching-adjusted Indirect Comparison of Troriluzole Versus Untreated Natural History Cohort in Spinocerebellar Ataxia (S35.009)
Beiner M, Powell L, Rogula B, Potashman M, Wirtz V, Schmahmann J, Perlman S, Coric V, L’Italien G. Matching-adjusted Indirect Comparison of Troriluzole Versus Untreated Natural History Cohort in Spinocerebellar Ataxia (S35.009). Neurology 2024, 102 DOI: 10.1212/wnl.0000000000205167.Peer-Reviewed Original ResearchDevelopment of a Novel Composite Measure (SCACOMS) to Assess Disease Progression in Spinocerebellar Ataxia (P7-3.019)
L’Italien G, Popoff E, Rogula B, Potashman M, Powell L, Beiner M, Coric V, Perlman S, Schmahmann J, Hendrix S. Development of a Novel Composite Measure (SCACOMS) to Assess Disease Progression in Spinocerebellar Ataxia (P7-3.019). Neurology 2024, 102 DOI: 10.1212/wnl.0000000000205169.Peer-Reviewed Original ResearchThe Phase 3 RESILIENT Study: Taldefgrobep Alfa in Spinal Muscular Atrophy (P2-11.008)
Lair L, Qureshi I, Bechtold C, Durham S, Campbell D, Marin J, Coric V. The Phase 3 RESILIENT Study: Taldefgrobep Alfa in Spinal Muscular Atrophy (P2-11.008). Neurology 2024, 102 DOI: 10.1212/wnl.0000000000205020.Peer-Reviewed Original ResearchNovel Bispecific Degrader BHV-1300 Achieves Rapid, Robust, and Selective IgG Reduction in Preclinical Models Including Nonhuman Primates (S43.004)
Bunin A, Lee S, Kazmierski W, McGrath K, Rossi A, Nicholson S, Dubowchik G, Dierks E, Sharpe N, Pirman D, Car B, Qureshi I, Coric V, Spiegel D. Novel Bispecific Degrader BHV-1300 Achieves Rapid, Robust, and Selective IgG Reduction in Preclinical Models Including Nonhuman Primates (S43.004). Neurology 2024, 102 DOI: 10.1212/wnl.0000000000206715.Peer-Reviewed Original ResearchPopulation Pharmacokinetic Modeling of Riluzole After Administration of a Next Generation Prodrug Troriluzole (S3.007)
Bertz R, Dumitrescu T, Wu Y, Jeong A, Sevinsky H, Qureshi I, Coric V. Population Pharmacokinetic Modeling of Riluzole After Administration of a Next Generation Prodrug Troriluzole (S3.007). Neurology 2024, 102 DOI: 10.1212/wnl.0000000000205212.Peer-Reviewed Original ResearchSafety, Tolerability, and Pharmacokinetics of Single and Multiple Rising Doses of a Next Generation Prodrug Troriluzole in Healthy Subjects (P4-4.012)
Sevinsky H, Awsare B, Rozakis R, Gentile K, Mydlow P, Ashbrenner E, Ham R, Stock D, Qureshi I, Coric V, Bertz R. Safety, Tolerability, and Pharmacokinetics of Single and Multiple Rising Doses of a Next Generation Prodrug Troriluzole in Healthy Subjects (P4-4.012). Neurology 2024, 102 DOI: 10.1212/wnl.0000000000205210.Peer-Reviewed Original ResearchAssociation of Anti-inflammatory Therapy Use with the Incidence of Parkinson’s Disease: A Person-Time Analysis Among Patients with Autoimmune Diseases (S2.003)
Haas J, Potashman M, Pandit A, Stafkey-Mailey D, Coric V, Singer W, L’Italien G. Association of Anti-inflammatory Therapy Use with the Incidence of Parkinson’s Disease: A Person-Time Analysis Among Patients with Autoimmune Diseases (S2.003). Neurology 2024, 102 DOI: 10.1212/wnl.0000000000204573.Peer-Reviewed Original Research
2023
Prognostic value of plasma biomarkers in a clinical trial of mild‐to‐moderate Alzheimer’s Disease
Qiu Y, Messer K, Jacobs D, Salmon D, Kaplita S, Wellington C, Stukas S, Askew B, Brewer J, Brody M, Donahue L, Drake J, Grossman K, Hendrix S, Jicha G, Leger G, Porsteinsson A, Shadyab A, Taylor C, Thomas R, van Dyck C, Zhang J, Coric V, Qureshi I, Feldman H, Group A. Prognostic value of plasma biomarkers in a clinical trial of mild‐to‐moderate Alzheimer’s Disease. Alzheimer's & Dementia 2023, 19 DOI: 10.1002/alz.076047.Peer-Reviewed Original ResearchModerate Alzheimer's diseaseAlzheimer's Disease Cooperative StudyPlasma NfLPlasma biomarkersCDR-SBADAS-cog11Baseline NfLAlzheimer's diseaseMRI outcomesClinical trialsTrial designBaseline biomarker measurementsBaseline plasma biomarkersPlacebo-controlled RCTsClinical trial designOnly significant associationGlobal functionClinical declineClinical outcomesP-tauTotal tauTreatment armsPrognostic valueSignificant treatment effectBiomarker changesP04 Taldefgrobep alfa: preclinical and clinical data supporting the phase 3 RESILIENT study in spinal muscular atrophy
Lair L, Qureshi I, Bechtold C, Heller L, Durham S, Campbell D, Marin J, Chen K, Coric V. P04 Taldefgrobep alfa: preclinical and clinical data supporting the phase 3 RESILIENT study in spinal muscular atrophy. Neuromuscular Disorders 2023, 33: s163. DOI: 10.1016/j.nmd.2023.07.381.Peer-Reviewed Original ResearchSpinal muscular atrophyMuscular atrophyClinical dataMuscle functionFlexor muscle functionMotor neuron lossRobust safety dataDownstream receptor signalingNon-ambulatory participantsAtrophic muscle fibersQuality of lifeSurvival motor neuron (SMN) proteinSMN upregulationMotor neuron geneNeuron lossMuscle weaknessSafety profileGastrocnemius weightPreclinical dataClinical studiesSafety dataMouse modelMuscle massNonclinical studiesMotor neuron protein