Treatment Trials

2 Clinical Trials for Various Conditions

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      COMPLETED
      PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
      Description

      The objective of this study is to evaluate the safety of long-term treatment with Kuvan.

      Conditions
      PhenylketonuriaHyperphenylalaninaemia
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      APPROVED_FOR_MARKETING
      Sapropterin Expanded Access Program
      Description

      The Purpose of this study is to provide patients with hyperphenylalaninemia (HPA) due to Phenylketonuria (PKU) access to sapropterin dihydrochloride and to collect more information about the safety of the drug in an expanded access program (EAP) until commercial product is available.

      Conditions
      Phenylketonuria
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