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
  • Discover the safety profile of eladocagene exuparvovec for these cases

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

Eladocagene exuparvovec is currently an investigational gene therapy product in the US. Eladocagene exuparvovec has been granted marketing authorization by the European Commission for the European Union Member States, Iceland, Norway, Northern Ireland, and Lichtenstein and granted marketing authorization by the UK MHRA for Great Britain.

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US-AADC-0566 | February 2024
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