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The effect of Ivacaftor in CF patients with a class III mutation

Primary objective is to objectively investigate the therapeutic potential of Ivacaftor in Dutch CF patients carrying a mutation associated with residual CFTR function. A secondary objective is to evaluate the correlations between individual…

A Multi Center, Prospective, Observational, Open-label, Pharmacokinetic Study of Tacrolimus in Heart and Lung Transplantation Patients during the First Days after Transplantation

Primary objective: To show that the variability of whole blood total and unbound plasma tacrolimus concentrations during the first 6 days post transplantation is larger than the variation of tacrolimus concentrations in stable clinical situation.…

Hypertonic saline in primary ciliary dyskinesia: a pilot study

We hypothesize that hypertonic saline nebulizations improve respiratory symptoms and quality of life in PCD patients.

Pharmacokinetics of tezacaftor-ivacaftor (Symkevi) and elexacaftor-tezacaftor-ivacaftor (Kaftrio) treatment in children with Cystic Fibrosis

This study aims to evaluate the PK profile and IIV of drug exposure in plasma in children (aged 6-17 years) receiving (elexacaftor-)tezacaftor-ivacaftor. Secondary objectives are to evaluate the relationship between covariates and PK parameters that…