PTC923-MD-003: A Study Drug for Patients with Phenylketonuria (PKU)
PKU (Phenylketonuria) is caused by a defective gene. When a person with PKU eats foods that contain protein, or eats aspartame, an artificial sweetener, a dangerous buildup of phenylalanine can occur. PTC923 is a study medication to reduce blood Phenylalanine (Phe) levels. This study will assess the safety and how effective this medication is for those of any age with a PKU diagnosis.
For more information contact:
| PI: Nicola Longo
| Department: PEDIATRIC GENETICS
| Approval Date: 2022-07-27 06:00:00
Specialties: Pediatric Genetics
Who can participate?
Age: All Ages
Volunteers: Volunteers with special conditions
Location: In Person
- Ages 0 years and older
- Diagnosis of PKU
- Participants of childbearing age must use specific birth control
- Problems with your stomach or intestines
- History of stomach surgery
Will I be paid for my time?