A Pilot Phase 2 Randomized Trial to Evaluate the Safety and Potential Efficacy of Etravirine in Friedreich Ataxia Patients

Background: A drug repositioning effort supported the possible use of the anti-HIV drug etravirine as a disease-modifying drug for Friedreich ataxia (FRDA). Etravirine increases frataxin protein and corrects the biochemical defects in cells derived from FRDA patients. Because of these findings, and...

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Bibliographic Details

Published in:	Children
Main Authors:	Gabriella Paparella, Cristina Stragà, Nicola Pesenti, Valentina Dal Molin, Gian Antonio Martorel, Vasco Merotto, Cristina Genova, Arianna Piazza, Giuseppe Piccoli, Elena Panzeri, Alessandra Rufini, Roberto Testi, Andrea Martinuzzi
Format:	Article
Language:	English
Published:	MDPI AG 2024-08-01
Subjects:	Friedreich ataxia etravirine treatment safety efficacy
Online Access:	https://www.mdpi.com/2227-9067/11/8/958