Open AccessDisease-Modifying Therapies for Multiple System Atrophy: Where Are We in 2022?
Author(s) -
Victoria Sidoroff,
Pam Bower,
Nadia Stefanova,
Alessandra Fanciulli,
Iva Stanković,
Werner Poewe,
Klaus Seppi,
Gregor K. Wenning,
Florian Krismer
Publication year - 2022
Publication title -
journal of parkinson's disease/journal of parkinson's disease (online)
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.747
H-Index - 45
eISSN - 1877-718X
pISSN - 1877-7171
DOI - 10.3233/jpd-223183
Subject(s) - disease , medicine , clinical trial , neuroinflammation , atrophy , neuroscience , bioinformatics , pathology , psychology , biology
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.