P356: Prediction of tissue frataxin levels with long term administration of nomlabofusp in adults with Friedreich’s ataxia using modeling and simulations

P356: Prediction of tissue frataxin levels with long term administration of nomlabofusp in adults with Friedreich’s ataxia using modeling and simulations, Clayton, Russell et al., Genetics in Medicine Open, Volume 3, 102321. doi: 10.1016/j.gimo.2025.102321 

Modeling and simulations using PK and PD data from short term studies can be used to predict a potential long term therapeutic dose. In the majority of patients with FRDA, daily administration of 50 mg nomlabofusp is predicted to result in skin frataxin concentrations that are ≥ 50% of concentrations observed in healthy controls.

P356: Prediction of tissue frataxin levels with long term administration of nomlabofusp in adults with Friedreich’s ataxia using modeling and simulations