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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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Principais autores: 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: Artigo
Sprog:Inglês
Udgivet: MDPI AG 2024-08-01
Serier:Children
Fag:
Friedreich ataxia
etravirine
treatment
safety
efficacy
Online adgang:https://www.mdpi.com/2227-9067/11/8/958
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