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…
• To assess feasibility and safety of a sequential treatment regime in which standard intensive chemotherapy (fludarabin-amsacrin-cytarabin) is directly followed by standard allogeneic stem cell transplantation (T cell depleted RIC alloSCT with…
Describe the electrocardiographic changes and areas of late myocardial activation encountered in PLN and PKP2 mutation carriers during ajmaline provocation and the relation of these differences to the development for early stages of arrhythmogenic…