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

ABSTRACT: 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 cure

SUBMITTER: Trantham SJ 

PROVIDER: S-EPMC10797190 | biostudies-literature | 2024 Mar

REPOSITORIES: biostudies-literature