Design: Variability of results limited utility of FXN protein measurement

The company noted, “FXN protein measurements in skeletal muscle had substantial overlap between FA patients and carriers and showed high intra-individual variability. More than half of FA patients had 25% or more variation in FXN protein levels between visits, with intra-individual coefficient of variation of 69%. The variability of results observed with this current method substantially limited the utility of FXN protein measurements to assess DT-216 pharmacology.”