Institut NeuroMyoGène
    Laboratoire Physiopathologie et Génétique du Neurone et du Muscle
    CNRS UMR 5261 -INSERM U1315
    Université de Lyon - Université Claude Bernard Lyon 1
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New publication team Puccio
Trends Mol Med 2024 : doi 10.1016/j.molmed.2023.12.002

Highlights

Omaveloxolone represents the first FDA-approved drug for Friedreich’s ataxia (FA).
Omaveloxolone targets nuclear factor erythroid 2-related factor 2 (NRF2), which is a master regulator in the antioxidant pathway.
The omaveloxolone clinical trial serves as an example for future design of clinical trials.
A resolutive cure for FA would probably be achieved only via combinatorial therapy.


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