Population Pharmacokinetic Modeling and Determination of Individual Exposure to Avalglucosidase Alfa in Adolescent and Adult Patients With Late-Onset Pompe Disease: Analysis of Pooled Data From Phase I to III Clinical Trials.
Gilles TuffalGilles TiraboschiFabrice HurbinPascale BoittetRachel PalmerJean-Marie MartinezDavid FabrePublished in: Therapeutic drug monitoring (2023)
A concatenated 3-compartment model was developed to describe the avalglucosidase alfa concentrations in patients with late-onset Pompe disease. None of the covariates tested could explain the interindividual variability.