Alain Ndayisaba, Ariana T Pitaro, Andrew S Willett, Kristie A Jones, Claudio Melo de Gusmao, Abby L Olsen, Jisoo Kim, Eero Rissanen, Jared K Woods, Sharan R Srinivasan, Anna Nagy, Amanda Nagy, Merlyne Mesidor, Steven Cicero, Viharkumar Patel, Derek H Oakley, Idil Tuncali, Katherine Taglieri-Noble, Emily C Clark, Jordan Paulson, Richard C Krolewski, Gary P Ho, Albert Y Hung, Anne-Marie Wills, Michael T Hayes, Jason P Macmore, Luigi Warren, Pamela G Bower, Carol B Langer, Lawrence R Kellerman, Christopher W Humphreys, Bonnie I Glanz, Elodi J Dielubanza, Matthew P Frosch, Roy L Freeman, Christopher H Gibbons, Nadia Stefanova, Tanuja Chitnis, Howard L Weiner, Clemens R Scherzer, Sonja W Scholz, Dana Vuzman, Laura M Cox, Gregor Wenning, Jeremy D Schmahmann, Peter Novak, Geoffrey S Young, Mel B Feany, Tarun Singhal, Vikram Khurana
Multiple system atrophy (MSA) is a fatal neurodegenerative disease of unknown etiology characterized by widespread aggregation of the protein alpha-synuclein in neurons and glia. Its orphan status, biological relationship to Parkinson's disease (PD), and rapid progression have sparked interest in drug development. One significant obstacle to therapeutics is disease heterogeneity. Here, we share our process of developing a clinical trial-ready cohort of MSA patients (69 patients in 2 years) within an outpatient clinical setting, and recruiting 20 of these patients into a longitudinal "n-of-few" clinical trial paradigm...
October 3, 2022: Cerebellum