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Disease-Modifying Therapies for Multiple System Atrophy: Where Are We in 2022?

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.

SUBMITTER: Sidoroff V

PROVIDER: S-EPMC9398078 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature

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Disease-Modifying Therapies for Multiple System Atrophy: Where Are We in 2022?

Sidoroff Victoria V Bower Pam P Stefanova Nadia N Fanciulli Alessandra A Stankovic Iva I Poewe Werner W Seppi Klaus K Wenning Gregor K GK Krismer Florian F

Journal of Parkinson's disease 20220101 5

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 comprehen ...[more]

PMID: 35491799

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