AADC deficiency resources
Access resources designed to support you and to share with caregivers following UpstazaTM 
(eladocagene exuparvovec) treatment for AADC deficiency.
‘What to expect’ guidance video
This video offers a unique insight into the post-treatment journey to support you in discussions with caregivers of patients who have received, or may receive, Upstaza.
‘Progress timeline’ video
‘A multidisciplinary approach to post-treatment care’ booklet
This booklet outlines the roles and responsibilities of the post-treatment multidisciplinary team (MDT) for Upstaza. This resource has been developed to support you in ensuring a co-ordinated approach to care is delivered.
‘Post-treatment care team’ guidance for caregivers
This leaflet is intended for caregivers, and clearly explains the roles and responsibilities of the MDT involved in post-treatment care.
Caregiver rehabilitation videos
These three instructional videos demonstrate how caregivers can support their loved ones to incorporate rehabilitation exercises into their everyday life.
Upstaza is indicated for the treatment of patients with a clinical, molecular, and genetically confirmed diagnosis of aromatic L-amino acid decarboxylase (AADC) deficiency with a severe phenotype, aged 18 months and older in the European Union Member States, Iceland, Liechtenstein, Norway, the United Kingdom, Israel, Taiwan, and Hong Kong, and paediatric patients aged 18 months and older in Brazil.
Registration conditions differ internationally; always consult local prescribing information and/or Summary of Product Characteristics before prescribing any product. For additional information, please contact PTC Therapeutics Medical Information at medinfo@ptcbio.com.
This medicine 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 at pharmacovigilance@ptcbio.com.
The website and materials have been developed by PTC Therapeutics for healthcare professionals only.
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Copyright © 2025 PTC Therapeutics. All rights reserved. Privacy Statement | Cookie Statement
GL-UPZ-0602 | July 2025
The website and materials have been developed by PTC Therapeutics for healthcare professionals only. Trademarks, registered or otherwise, are the property of their respective owner(s).
Copyright © 2025 PTC Therapeutics. All rights reserved. Privacy Statement | Cookie Statement
GL-UPZ-0602 | July 2025
