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 dataRe-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 ResearchUntreated patientsDisease progressionDevelopment 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 ResearchAssess disease progressionDisease progression