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CNS Drugs
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Open Access 08-08-2024 | Disease of the Nervous System in Children | Review Article

New and Emerging Drug and Gene Therapies for Friedreich Ataxia

Authors: Varlli Scott, Martin B. Delatycki, Geneieve Tai, Louise A. Corben

Published in: CNS Drugs | Issue 10/2024

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Abstract

The life shortening nature of Friedreich Ataxia (FRDA) demands the search for therapies that can delay, stop or reverse its relentless trajectory. This review provides a contemporary position of drug and gene therapies for FRDA currently in phase 1 clinical trials and beyond. Despite significant scientific advances in the specificity of both compounds and targets developed and investigated, challenges remain for the advancement of treatments in a limited recruitment population. Currently therapies focus on reducing oxidative stress and improving mitochondrial function, modulating frataxin controlled metabolic pathways and gene replacement and editing. Approval of omaveloxolone, the first treatment for individuals with FRDA aged 16 years and over, has created much excitement for both those living with FRDA and those that care for them. The process of approval of omaveloxolone by the US Food and Drug Administration highlighted the importance of sensitive outcome measures and the significant role of data from natural history studies.
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Metadata
Title
New and Emerging Drug and Gene Therapies for Friedreich Ataxia
Authors
Varlli Scott
Martin B. Delatycki
Geneieve Tai
Louise A. Corben
Publication date
08-08-2024
Publisher
Springer International Publishing
Published in
CNS Drugs / Issue 10/2024
Print ISSN: 1172-7047
Electronic ISSN: 1179-1934
DOI
https://doi.org/10.1007/s40263-024-01113-z

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