Perspectives of the Friedreich ataxia community on gene therapy clinical trials

Shandra J. Trantham,Mackenzi A. Coker,Samantha Norman,Emma Crowley,Julie Berthy,Barry J. Byrne,Sub Subramony,XiangYang Lou,Manuela Corti; Molecular Therapy Methods & Clinical Development, Volume 32, Issue 1, 101179, doi:10.1016/j.omtm.2023.101179 

 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.