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

Ultragenyx Pharmaceutical Inc · 2019
ClinicalTrials.gov · Datasets · License: Public Domain · 2019
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gene-therapygenetic-diseases
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.
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