Precision medicine and Friedreich ataxia: promoting equity, beneficence, and informed consent for novel gene therapies

Kwa, F., Kendal, E. Precision medicine and Friedreich ataxia: promoting equity, beneficence, and informed consent for novel gene therapies. Int J Equity Health 23, 230 (2024). doi:10.1186/s12939-024-02318-w 

This article will use FA as an example to explore some of the practical and ethical issues emerging in precision medicine for rare diseases. It will first describe the existing management strategies available for FA patients, before considering the potential impact of gene therapy trials on the prevention and treatment of disease symptoms. Finally, ethical considerations will be discussed, including equity of access and managing resource allocation dilemmas; balancing benefits, burdens and harms; and gaining informed consent for novel treatments.