Safety, pharmacokinetics, and pharmacodynamics of nomlabofusp (<scp>CTI</scp>‐1601) in Friedreich's ataxia

Abstract

Results from this study support a favorable safety profile for nomlabofusp. Subcutaneous nomlabofusp injections were quickly absorbed; higher doses and daily administration resulted in increased tissue frataxin concentrations. Future studies will evaluate longer-term safety and possible efficacy of nomlabofusp.