2 results
Approved WMORecruiting
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…
Approved WMOCompleted
This non-controlled multicentric phase II study is designed to assess the safety and to describe (in relation to children of higher age) the pharmacodynamics of recombinant ASNase for first-line treatment of infants (< 1 year of age at…