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.

Perspectives of the Friedreich ataxia community on gene therapy clinical trials