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The primary objectives of this open-label study were to assess the pharmacodynamics of eladocagene exuparvovec and the safety of the SmartFlow magnetic resonance (MR)-compatible ventricular cannula for administering eladocagene exuparvovec in pediatric participants with l-amino acid decarboxylase (AADC) deficiency.
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Pearl et al. (2024) studied this question.
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