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.