Tissue frataxin concentration data from these studies are consistent with prior studies demonstrating that lower frataxin concentrations are associated with earlier onset of disease. The range of characteristics observed in the population of patients with FRDA participating in the nomlabofusp interventional studies is representative of the general FRDA adult population. Buccal and skin cell frataxin levels correlate with each other.
Monday, March 17, 2025
P289: Disease characteristics and tissue frataxin concentrations in adults with Friedreich’s ataxia participating in nomlabofusp interventional studies
Russell Clayton, Mohamed Hamdani, Noreen Scherer,, P289: Disease characteristics and tissue frataxin concentrations in adults with Friedreich’s ataxia participating in nomlabofusp interventional studies,
Genetics in Medicine Open, Volume 3, Supplement 2, 2025, 102254, ISSN 2949-7744, doi10.1016/j.gimo.2025.102254.
