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Long-term safety and efficacy of sapropterin: The PKUDOS registry experience

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Evidence- and consensus-based recommendations for the use of pegvaliase in adults with phenylketonuria

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Real-world treatment, dosing, and discontinuation patterns among patients treated with pegvaliase for phenylketonuria: Evidence from dispensing data

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Use of pegvaliase in the management of phenylketonuria: Case series of early experience in US clinics

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Pegvaliase for the treatment of phenylketonuria: Results of a long-term phase 3 clinical trial program (PRISM)

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Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial

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