3 results
Approved WMORecruiting
Primary objectives: To evaluate the efficacy of long-term prophylactic treatment with PHA-022121 in preventing breakthrough angioedema attacks in patients with AAE-C1-INH. Secondary objectives: To evaluate the safety of long-term prophylactic…
Approved WMORecruiting
Investigating the long-term safety of personalized cholic acid treatment in patients with defects in bile acid synthesis based on clinical and biochemical parameters.
Approved WMORecruiting
To determine RD of BLU-263To assess if treatment with BLU-263 improves outcomes compared to placebo + BSC, as assessed using the ISM-SAFTo assess the long-term safety and tolerability of treatment with BLU-263To assess the long-term efficacy of…