3 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 aim of our project is to perform a novel mass screening to detect CD in 12 months-4 years old children who visit the YHCCs in a well-described region in the Netherlands, to show that it is feasible, efficient, cost-effective and well accepted by…
The primary objectives of this study are: - To evaluate the efficacy of GFT505 80mg and GFT505 120mg once daily (according to adaptive design, after 6-month interim safety analysis of the 80mg data on at least 50% of patients) for 52 weeks versus…