Long‐term safety of sapropterin in paediatric and adult individuals with phenylalanine hydroxylase deficiency: Final results of the Kuvan® Adult Maternal Paediatric European Registry multinational observational study

Abstract Phenylketonuria is a rare inherited disorder that disrupts the metabolism of phenylalanine (Phe) to tyrosine by phenylalanine hydroxylase (PAH). Sapropterin dihydrochloride (Kuvan®) is approved for use in Europe to reduce blood Phe levels and improve Phe tolerance in sapropterin‐responsive...

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Bibliographic Details
Published in:Journal of inherited metabolic disease 2024-09
Main Authors: Feillet, François, Arnoux, Jean‐Baptiste, Delgado, María Bueno, Burlina, Alberto, Chabrol, Brigitte, Kucuksayrac, Ece, Lagler, Florian B., Muntau, Ania C., Olsson, David, Paci, Sabrina, Rutsch, Frank, van Spronsen, Francjan J.
Format: Article
Language:English
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Summary:Abstract Phenylketonuria is a rare inherited disorder that disrupts the metabolism of phenylalanine (Phe) to tyrosine by phenylalanine hydroxylase (PAH). Sapropterin dihydrochloride (Kuvan®) is approved for use in Europe to reduce blood Phe levels and improve Phe tolerance in sapropterin‐responsive individuals. KAMPER (NCT01016392) is an observational, multinational registry assessing long‐term safety and efficacy of sapropterin. Five hundred and seventy‐six participants with PAH deficiency were enrolled from nine European countries (69 sites; December 2009–May 2016). Participants were aged
ISSN:0141-8955
1573-2665
1573-2665
DOI:10.1002/jimd.12796