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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
Eladocagene Exuparvovec is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC Therapeutics at pharmacovigilance@ptcbio.com.

Registration conditions differ internationally; always consult local prescribing information and/or Summary of Product Characteristics before prescribing any products.

MED-ALL-PTC-AADC-2500006 | September 2025

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

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MED-ALL-AADC-2300018 | June 2025

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