2 results
Approved WMORecruiting
To assesses the efficacy of Tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1.
Approved WMOCompleted
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…