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 WMORecruiting
Primary Objectives:• To assess the safety and tolerability of DCC 3014.• To characterize the pharmacokinetic (PK) profile of DCC 3014.• To determine the maximum tolerated dose (MTD) of DCC 3014.• To determine the recommended Phase 2 dose (RP2D) of…