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KAMPER: Kuvan (BH4) in phenylketonuria
Manage episode 456613899 series 3001064
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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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