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Enzyme substitution therapy
Pegvaliase for Phenylketonuria
Recruiting2 awardsPhase 3
Gainesville, Florida
This trial is testing pegvaliase injections in adolescents with PKU. The goal is to see if these injections are safe and effective. Pegvaliase helps the body break down a harmful substance that people with PKU cannot process on their own. Pegvaliase is a newer treatment for PKU, following earlier treatments like dietary restriction and sapropterin.
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