3 results
Primary objectives: to evaluate the efficacy of three different single doses of PHA-022121 versus placebo in achieving angioedema symptom relief during acute attacks and the efficacy of prophylactic treatment with PHA-022121 versus placebo in…
Primary ObjectivesThe primary objectives of this study are as follows:• To evaluate the safety and tolerability of risdiplam.• To investigate the pharmacokinetics (PK) of risdiplam and metabolites as appropriate.Secondary ObjectiveThe secondary…
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…