2024
The ALSFRS-R Summit: a global call to action on the use of the ALSFRS-R in ALS clinical trials
Genge A, Cedarbaum J, Shefner J, Chio A, Al-Chalabi A, Van Damme P, McDermott C, Glass J, Berry J, van Eijk R, Fournier C, Grosskreutz J, Andrews J, Bertone V, Bunte T, Couillard M, Cummings C, Kittle G, Polzer J, Salmon K, Straub C, van den Berg L. The ALSFRS-R Summit: a global call to action on the use of the ALSFRS-R in ALS clinical trials. Amyotrophic Lateral Sclerosis And Frontotemporal Degeneration 2024, 25: 382-387. PMID: 38396337, DOI: 10.1080/21678421.2024.2320880.Peer-Reviewed Original ResearchAmyotrophic Lateral Sclerosis Functional Rating ScaleALSFRS-RClinical trialsStatistical analysis planMonitor functional changesRating ScaleOutcome measuresFunctional Rating ScalePrimary outcome measureClinical trial designALS clinical trialsFunctional changesTrial designTrialsClinical trialistsScaleALS trials
2021
Seeking progress in disease modification in Parkinson disease
Lungu C, Cedarbaum J, Dawson T, Dorsey E, Faraco C, Federoff H, Fiske B, Fox R, Goldfine A, Kieburtz K, Macklin E, Matthews H, Rafaloff G, Saunders-Pullman R, Schor N, Schwarzschild M, Sieber B, Simuni T, Surmeier D, Tamiz A, Werner M, Wright C, Wyse R. Seeking progress in disease modification in Parkinson disease. Parkinsonism & Related Disorders 2021, 90: 134-141. PMID: 34561166, DOI: 10.1016/j.parkreldis.2021.09.006.Peer-Reviewed Original ResearchConceptsDisease modificationParkinson's diseaseDisease-modifying benefitsNovel trial designsDrug therapyPatient populationTrial failuresTherapeutic targetTrial designNeurological disordersRelevant biomarkersTherapeutic developmentNational InstituteDiseaseReview articleResearch prioritiesKey stakeholder groupsFailureLikelihood of successTherapyStrokeBiomarkers