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  1. 1
    Patient selection considerations for AADC deficiency gene therapy

    Patient selection considerations for AADC deficiency gene therapy by Agathe Roubertie, Irina Anselm, Bruria Ben‐Zeev, Wuh‐Liang Hwu, Ashutosh Kumar, Berrin Monteleone, Shin‐ichi Muramatsu, Vincenzo Leuzzi, Salvador Ibáñez, Scellig Stone, Phillip L. Pearl

    Published 2024-03-01
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  2. 2
    O14: Administration of eladocagene exuparvovec using the SmartFlow magnetic resonance-compatible ventricular cannula in pediatric patients with aromatic ʟ-amino acid decarboxylase deficiency

    O14: Administration of eladocagene exuparvovec using the SmartFlow magnetic resonance-compatible ventricular cannula in pediatric patients with aromatic ʟ-amino acid decarboxylase... by Alexis Krolick, Paul Hwu, Donald Gilbert, Bruria Ben-Zeev, Daniel Curry, Scellig Stone, Matthew Vestal, Christian Werner, Vinay Penematsa, Antonia Wang, Rezwanur Rahman, Lee Golden, Phillip Pearl

    Published 2025-01-01
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