Latest News


PTC Therapeutics; Eladocagene exuparvovec (Upstaza)  

The European Medical Association (EMA) has recommended granting a marketing authorisation in the European Union (EU) for Upstaza (eladocagene exuparvovec), a therapy for the treatment of adult and paediatric patients with  a confirmed diagnosis of AADCd.


Their recommendation will now be forwarded to the European Commission for the adoption of a decision on the EU-wide marketing authorisation.

The Trust awaits the announcement of a decision from the European Commission and we will post updates to our community as we get them.

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To read the full article please click on the image ...

The summary of opinion from the EMA ...

Summary of opinion EMA
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Gene Therapy Updates ...
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NICE (National Institute for Health and Care Excellence) has invited The AADC Research Trust to participate in the next stage of the appraisal of the Eladocagene exuparvovec (Upstaza) for the treatment of Aromatic Amino Acid Decarboxylase deficiency.


This evaluation stage will culminate in a committee meeting due to be held in November 2022. In preparation for this we are conducting a 'Patient Experience Survey' and 'Patient Insight Interviews'. The data collected from these will be collated and summarised for submission to NICE. 

If you would like to participate please contact Julie;


The Trust is excited to participate in bringing another gene therapy treatment to our AADCd children. We will keep you updated with any further developments.

Data published by Professor Krystof Bankiewicz; AAV2-AADC Gene Therapy to the Mid-brain ...

Professor Krystof Bankiewicz and Dr Toni Pearson have published data on their trial; AAV2-AADC Gene Therapy to the Substansia Nigra Pars Compacta & Ventral Tegmental area of the brain.

The open access article is titled 'Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons.' It is a comprehensive study, covering an age range of 4 - 26 years in age and various AADC mutation combinations. The outcomes are clearly positive with a cessation of Oculogyric Crisis, the most common AADCd symptom, in almost all patients.

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More AADCd Children receive Gene Therapy in Poland & USA ...

2020 and 2021 have brought many challenges with the emergence of COVID-19. Many aspects of our lives have been put on hold. 


Essential surgeries, including AAV2-hAADC Gene Therapy Treatment, had to be postponed due to the extremely difficult circumstances posed by the threat of COVID.


However, Professor Bankiewicz and his incredible team have gone to extraordinary lengths to meet that challenge...


The AADC Trust is thrilled to share the wonderful news that three more AADCd children have received Gene Therapy. Two received treatment in Poland and one received treatment in the USA ...

Meet 11 year old Julia from Norway.

Julia is the 21st AADCd child to receive

AAV2-hAADC Gene Replacement Therapy.

Meet 10 year old Paul from Germany.

Paul is the 22nd AADCd child to receive

AAV2-hAADC Gene Replacement Therapy.

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Meet 6 year old Jamell from the USA.

Jamell is the 23rdd AADCd child to receive

AAV2-hAADC Gene Replacement Therapy.