How great a risk do you take? A qualitative study exploring attitudes of individuals with Friedreich ataxia towards gene therapy

Scientists and pharmaceutical companies are working towards delivering gene therapy for Friedreich ataxia (FRDA). Understanding the views of people with lived experience of FRDA and their parents toward gene therapy is essential to inform trial design and identify potential barriers to participation in clinical trials. The goals of this study were to identify the attitudes towards gene therapy held by individuals with FRDA and parents of individuals with FRDA, and to explore how these may impact future trials for this condition. Audio-recorded, semi-structured, qualitative interviews with nineteen Australians explored experiences of FRDA, knowledge about clinical trials, views on gene therapy including risks and benefits, and potential barriers to participation in trials. Participants included thirteen individuals living with FRDA aged between 15-43 years, and six parents of children with FRDA aged 4-12 years of age. Thematic analysis of the interviews identified six main themes. Findings from this study indicate there is strong desire for information regarding gene therapy in FRDA however the current level of uncertainty around gene therapy makes decision making challenging. The desire to maintain functional status and avoid additional risk of deterioration from an investigational treatment was apparent. Importantly, neurological targets were identified as preferred for gene therapy trials. Further research is required to identify if attitudes and perceptions differ according to geographical location and disease stage.

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