3 results
Approved WMORecruiting
the primary objective of our study is to define a set of patient characteristics that can predict the effectiveness of an MRA in treating symptomatic OSA. The aim of our study is to develop a prediction model to calculate the success rate in an…
Approved WMORecruiting
Phase 2:Primary ObjectivesTo determine the recommended Phase3 dose of mitapivat by evaluating the effect of 2 dose levels of mitapivat versus placebo on:• Anemia in subjects with sickle cell disease (SCD)• SafetySecondary ObjectivesTo evaluate the…
Approved WMORecruiting
The primary objective of this study is to evaluate the efficacy of olipudase alfa in adult patients with acid sphingomyelinase deficiency.