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Review
. 2022 Feb;22(2):101-114.
doi: 10.1080/14737175.2022.2029703. Epub 2022 Feb 10.

Current and emerging treatment modalities for spinocerebellar ataxias

Shaila D Ghanekar  1   2 , Sheng-Han Kuo  3   4 , Joseph S Staffetti  1   2 , Theresa A Zesiewicz  1   2
Affiliations
Review

Current and emerging treatment modalities for spinocerebellar ataxias

Shaila D Ghanekar et al. Expert Rev Neurother. 2022 Feb.

Abstract

Introduction: Spinocerebellar ataxias (SCA) are a group of rare neurodegenerative diseases that dramatically affect the lives of affected individuals and their families. Despite having a clear understanding of SCA's etiology, there are no current symptomatic or neuroprotective treatments approved by the FDA.

Areas covered: Research efforts have greatly expanded the possibilities for potential treatments, including both pharmacological and non-pharmacological interventions. Great attention is also being given to novel therapeutics based in gene therapy, neurostimulation, and molecular targeting. This review article will address the current advances in the treatment of SCA and what potential interventions are on the horizon.

Expert opinion: SCA is a highly complex and multifaceted disease family with the majority of research emphasizing symptomatic pharmacologic therapies. As pre-clinical trials for SCA and clinical trials for other neurodegenerative conditions illuminate the efficacy of disease modifying therapies such as AAV-mediated gene therapy and ASOs, the potential for addressing SCA at the pre-symptomatic stage is increasingly promising.

Keywords: Spinocerebellar ataxia; gene therapy; neurodegenerative diseases; neurostimulation; treatment.

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Figures

Figure 1.
Figure 1.
Gene therapy for SCA.
See this image and copyright information in PMC

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