A Pilot Phase 2 Randomized Trial to Evaluate the Safety and Potential Efficacy of Etravirine in Friedreich Ataxia Patients.
Gabriella PaparellaCristina StragàNicola PesentiValentina Dal MolinGian Antonio MartorelVasco MerottoCristina GenovaArianna PiazzaGiuseppe PiccoliElena PanzeriAlessandra RufiniRoberto TestiAndrea MartinuzziPublished in: Children (Basel, Switzerland) (2024)
In this open trial etravirine treatment was safe, reasonably well tolerated and appreciably improved neurological function and exercise performance. Even though a placebo effect cannot be ruled out, these results suggest that etravirine may represent a potential therapeutic agent in FRDA deserving testing in a randomized placebo-controlled clinical trial.