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Latest News

As promised we bring you the latest updates from around the world as they happen.

 

PTC Biotherapeutic's gene therapy drug Upstaza has been granted license for use in the European Union, for the treatment of Aromatic Amino Acid Decarboxylase deficiency. 

 

Looking ahead, it is important to remember that all AADCd children share a mild, moderate or severe form of this complex disease, based on mutations and remain biochemically serotonin deficient after gene therapy treatment.

 

Treating only the dopamine pathway means we must remain vigilant regarding autonomic dysfunction and other non dopamine related symptoms since the discrepancy between these two chemicals changes post treatment. We look forward to the longitudinal data, following our European children, after receiving gene therapy and pray for a continuous positive response.

 

We are grateful science is advancing and that, over the past 17 years, The AADC Research Trust has had the opportunity to be an important part of driving this and other innovative gene therapy treatments forward.

 

We remain hopeful, that one day, we can finally say AADCd is completely cured.

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

Gene Therapy Updates ...
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The AADC Research Trust is working closely with NICE (National Institute for Health and Care Excellence) to complete the appraisal of eladocagene exuparvovec (Upstaza), a gene therapy drug for the treatment of Aromatic Amino Acid Decarboxylase deficiency.

 

The Trust has completed its submissions and is eagerly awaiting the next stage in anticipation of approval for the drug to be available in the UK, following its approval for use in the EU earlier this year. In November 2022 we will be joining the technology appraisal committee meeting to review the evaluation. 

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.

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