We've found 4 total results for "Phenylketonurias".

A Study of PTC923 in Participants With Phenylketonuria

The main purpose of this trial is to evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in participants with phenylketonuria as measured by mean change in blood Phe levels from baseline to Weeks 5 and 6 (that is, the average of each respect...

Age: Birth - 66+

Gender: All

Gene Therapy Clinical Study in Adult PKU

This is a Phase 1/2, open-label, randomized, concurrently-controlled, dose escalation study to evaluate the safety and efficacy of HMI-102 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of HMI-102 and will be followed fo...

Age: 18 - 55 years

Gender: All