A Pilot Phase 2 Randomized Trial to Evaluate the Safety and Potential Efficacy of Etravirine in Friedreich Ataxia Patients
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.
Authors
- Nicola Pesenti (ORCID: https://orcid.org/0000-0002-2475-7927)
- Arianna Piazza
- Andrea Martinuzzi (ORCID: https://orcid.org/0000-0002-0319-3579)
- Elena Panzeri
- Roberto Testi (ORCID: https://orcid.org/0000-0001-5925-015X)
- Alessandra Rufini (ORCID: https://orcid.org/0000-0001-5654-9615)
- Gabriella Paparella (ORCID: https://orcid.org/0000-0002-9986-9907)
- Cristina Stragà (ORCID: https://orcid.org/0009-0005-9756-0534)
- Valentina Dal Molin
- Gian Antonio Martorel
- Vasco Merotto
- Cristina Genova
- Giuseppe Piccoli
Institutions
- University of Rome Tor Vergata (IT)
- IRCCS Eugenio Medea (IT)
- University of Milano-Bicocca (IT)
Publication Details
- Journal
- Children
- Published
- 2024-08-09
- DOI
- https://doi.org/10.3390/children11080958
- Citations
- 2
- Primary Topic
- Genetic Neurodegenerative Diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.54
Funders
- Ministero della Salute