Department of Neurology, Medical University ofInnsbruck, Innsbruck, Austria
| [b] The Multiple SystemAtrophy Coalition, Inc., McLean, VA USA | [c]
NeurologyClinic, University Clinical Center of Serbia, School of Medicine, University of Belgrade, Serbia
Correspondence to: Florian Krismer, MD, PhD, and Gregor K. Wenning, MD, PhD, MSc, Department of Neurology, Medical University Innsbruck, Anichstrasse 35, A-6020 Innsbruck, Austria. Tel.: +43 512 504 80932 /+43 512 504 81811; E-mails: [email protected] / [email protected].
Note:  These authors contributed equally to this work.
Abstract: Multiple system atrophy is a rapidly progressive and fatal neurodegenerative disorder. While numerous preclinical studies suggested efficacy of potentially disease modifying agents, none of those were proven to be effective in large-scale clinical trials. Three major strategies are currently pursued in preclinical and clinical studies attempting to slow down disease progression. These target α-synuclein, neuroinflammation, and restoration of neurotrophic support. This review provides a comprehensive overview on ongoing preclinical and clinical developments of disease modifying therapies. Furthermore, we will focus on potential shortcomings of previous studies that can be avoided to improve data quality in future studies of this rare disease.