Emerging therapies for Friedreich Ataxia and the prospect of future combination treatments

2025/09/26 by Jarmon G. Lees, Li Li, Shiang Y. Lim · 1 voice
Biochemistry, Genetics and Molecular Biology · Neuroscience · #Genetic Neurodegenerative Diseases #Mitochondrial Function and Pathology #Neuroscience and Neuropharmacology Research

paper · pdf · doi:10.1080/23995270.2025.2563497

openalex publication_date 2025/09/26 · openalex created_date 2025/10/10 · openalex updated_date 2026/07/28

Abstract

1. Friedreich ataxia (FRDA) is a rare, autosomal recessive neurodegenerative disorder marked by progressive nervous system degeneration and multisystem involvement, including cardiac, musculoskelet...

Citations

• AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich’s ataxia

Discussions

• Spotlighting Li for #ECIExcellence2025! ✨ Her first publication is out, an editorial on emerging therapies for Friedreich ataxia: doi.org/10.1080/2399... Now beginning her PhD on heart disease in Frie [bsky, 1 points, 0 comments]

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