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Home | Central nervous system | Aromatic L-amino acid decarboxylase (AADC) deficiency | Publications | Publication: Intraputaminal gene delivery in two patients with aromatic L-amino acid decarboxylase deficiency

Publication: Intraputaminal gene delivery in two patients with aromatic L-amino acid decarboxylase deficiency

This case study published in Movement Disorders describes the outcomes of 2 patients with aromatic L-amino acid decarboxylase (AADC) deficiency treated with intraputaminal gene therapy

  • Learn more about intraputaminal gene therapy for AADC deficiency, a rare autosomal recessive disease
  • Review the key efficacy outcomes of eladocagene exuparvovec treatment in 2 patients over 10 years of age with severe forms of AADC deficiency
  • Understand the safety profile of eladocagene exuparvovec for these cases
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MED-US-CORP-2500008 | November 2025

François-Heude MC, et al. Mov Disord Clin Pract. 2023 Feb 24;10(5):811-818.

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