Phenylketonuria therapy options mature past low-phenylalanine diet
Pegvaliase commercial maturity, sapropterin use, and emerging gene therapy programs are restructuring phenylketonuria management.
Phenylketonuria has been a low-phenylalanine-diet category for decades, with sapropterin available for responsive patients. Pegvaliase (an enzyme substitution therapy) has commercial maturity in adults with elevated blood phenylalanine despite optimised therapy, and AAV gene therapy programs delivering a working PAH gene are reaching pivotal data. The category has gone from diet-only to a real pharmacotherapy frame in adults.
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