TrialOrphanet journal of rare diseases2021

Long-term efficacy and safety of sapropterin in patients who initiated sapropterin at < 4 years of age with phenylketonuria: results of the 3-year extension of the SPARK open-label, multicentre, randomised phase IIIb trial.

Ania C Muntau et al.PubMed ↗Full text ↗Publisher ↗

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