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Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

Ultragenyx Pharmaceutical Inc · 2019
ClinicalTrials.gov · Datasets · License: Public Domain · 2019
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gene-therapy
Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein
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