2 results
Approved WMORecruiting
To compare tisagenlecleucel treatment strategy to SOC treatment strategy with respect to delaying the composite event of disease progression / stable disease at or after the week 12 assessment; or death at any time.
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…