Perspectives of the Friedreich ataxia community on gene therapy clinical trials.

Gene therapy is a potential treatment for Friedreich ataxia, with multiple programs on the horizon. The purpose of this study was to collect opinions about gene therapy from individuals 14 years or older with Friedreich ataxia or parents/caregivers of Friedreich ataxia patients who were diagnosed as children 17 or younger. Participants were asked to complete a survey after reading brief educational materials regarding gene therapy. Most of the patients captured in this survey have an early-onset (classical) presentation of the disease. Participants expressed urgency in participating in gene therapy clinical trials despite the associated risks. About half of the respondents believed that gene therapy would cease progression or minimize symptoms, whereas nearly one-fourth expected to be cured. The survey also revealed how participants perceive their symptom burden, because a substantial majority reported that balance/walking issues most interfere with their quality of life and would be the symptom they would prioritize treating. Although not statistically significant, more caregivers prioritized treating cardiomyopathy than patients. This study provides valuable information on priorities, beliefs, and expectations regarding gene therapy and serves to guide future gene therapy opinion studies and gene therapy trial design.

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© 2023 The Author(s).

Déclaration de conflit d'intérêts

M.C. and B.J.B. are co-founders of AavantiBio, which supported the employee at ClinicalMind who provided graphical support for the educational material written by the study team. The survey was completed before the acquisition of AavantiBio by Solid Biosciences. None of the authors have any financial, commercial, legal, or professional relationship with Solid Biosciences. There was no commercial support for this study. The study was developed and conducted by the academic team at the University of Florida. Thus, the authors declare that they have no competing interests.