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

• Main Authors: Gabriella Paparella (Author), Cristina Stragà (Author), Nicola Pesenti (Author), Valentina Dal Molin (Author), Gian Antonio Martorel (Author), Vasco Merotto (Author), Cristina Genova (Author), Arianna Piazza (Author), Giuseppe Piccoli (Author), Elena Panzeri (Author), Alessandra Rufini (Author), Roberto Testi (Author), Andrea Martinuzzi (Author)
• Format: Book
• Published: MDPI AG, 2024-08-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...