KAMPER: Kuvan (BH4) in phenylketonuria
Dec 20, 2024•26 min
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Episode description
Dr François Feillet returns to the podcast to discuss the final results of the KAMPER study, evaluating the long-term safety of sapropterin in phenylketonuria (phenylalanine hydroxylase deficiency).
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
François Feillet, et al
https://doi.org/10.1002/jimd.12796
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