Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

This study is testing a gene therapy for the heart problems caused by Friedreich's Ataxia — a rare genetic disease that damages the nervous system and heart. The therapy uses a virus (AAV vector) to deliver a healthy copy of the frataxin gene to heart muscle cells. You may be eligible if... - You are between 12 and 50 years old - You have a confirmed diagnosis of Friedreich's Ataxia (with the GAA expansion in both alleles) - You have evidence of heart disease related to Friedreich's Ataxia - Your heart ejection fraction is between 35% and 75% - Your organ function (liver, kidneys, blood counts) meets minimum thresholds You may NOT be eligible if... - You are taking immunosuppressive medications - You have uncontrolled diabetes (HbA1c over 7%) - You have decompensated heart failure (NYHA class III-IV) - You have had any cancer in the past 5 years - You have previously received any gene or cell therapy - You are pregnant or breastfeeding - You have obstructive coronary artery disease or had a recent blood clot Talk to your doctor to see if this trial is right for you.