3 results
To characterize safety, tolerability, pharmacodynamic effects, and pharmacokinetics of the oral sGC stimulator BAY 1021189 in addition to standard therapy for heart failure with reduced EF (HFrEF) over 12 weeks in patients with worsening chronic…
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 characterize the PK of eltrombopag at steady state after oral administration in pediatric patients with SAA.Secondary (key only, see protocol page 40-41 for all objectives): Safety and tolerability. Efficacy (overall response rate ORR).