Treatment Trials

1 Clinical Trials for Various Conditions

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      COMPLETED
      Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia
      Description

      The primary objective of this study is to determine the long-term safety of DTX401 following a single intravenous (IV) dose in adults with GSDIa.

      Conditions
      Glycogen Storage Disease Type IAVon Gierke's Disease (GSD Type Ia)
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