4 results
the objective of the current study is to evaluate the safety and efficacy of enzyme therapy in the Dutch population of patients with MPS I, II and VI; to compare this to the natural course of these diseases; to make an inventory of the direct and…
The purpose of the study is to investigate how quickly and to what extent flurbiprofen is absorbed and eliminated from the body (this is called pharmacokinetics) when it is administered via a FTS. Furthermore, the pharmacokinetics of FTS will be…
Objectives The main objective of the FAIR-PARK II trial is to demonstrate an effect of DFP on the course of PD (including both disease-modifying and symptomatic effects). The trial's overall objective can be summarized as follows: to…
Studying the effect of deferiprone on the motor function of patients with Pelizaeus-Merzbacher disease.