4 results
To assess the efficacy of ravulizumab versus placebo in the treatment of adult and adolescent participants with HSCT-TMA.
To compare the efficacy and tolerability of a combination therapy with ursodeoxycholic acid (12-16 mg/kg body weight plus budesonide (9mg/d) vs. ursodeoxycholic acid (12-16 mg/kg BW/d) plus placebo in the treatment of PBC. To study safety and…
PrimaryTo assess the efficacy of ravulizumab in the treatment of participants with TMASecondaryTo characterize TMA responseTo assess impact on hemoglobin levels To evaluate change in kidney function To assess duration of Complete TMA Response and…
Primary:• To evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria (PKU) as measured by mean change in blood Phe levels from baseline to Weeks 5 and 6 (ie, the average of each respective…