The AADC Trust is launching the #oneRAREstep Campaign to raise funds for our AADCd children to access life saving and life transforming pioneering brain delivered AAV2-hAADC Gene Therapy Treatment. This surgery is setting a benchmark for future treatments and has the potential to help many other children suffering from diseases with single gene defects...
Click on the image to go to the #oneRAREstep Campaign Just Giving Page...
AAV2-hAADC Gene Replacement Therapy has already shown incredible results and provided substantial relief from complex life-threatening symptoms for several AADCd children To view some of their stories click on the links below...
Audrey's Gene Therapy Journey
It is life changing experience"
Kacper's Gene Therapy Journey
"Children have a chance for a new, a better life. I would like for all children with this disease to be able to receive GT treatment and live as close to a normal life as possible, so they are not chained to strollers or beds"
Ela (Kacper's mum)
Alex's Gene Therapy Journey
"The quality of life improvement for the child is immeasurable and it offers hopes to the child as they continue to improve and develop after the GT"
Ashlee (Alex's mum)
This video published in 2019 by The Columbus Children's Foundation, titled "Amazing advances in the treatment of childhood Parkinson's" showcases the amazing progress of AADCd children, following AAV2-hAADC Gene Replacement Therapy Treatment...
A word from The Managing Director
“Recently I have had the extreme privilege of witnessing 'first-hand' a spectacular event, a truly surreal personal experience and one I will never forget!
Professor Manju Kurian, Consultant Paediatric Neurologist at Great Ormond Street Hospital and myself Lisa Flint, Founder and Managing Director of the AADC Trust, travelled to Poland and had the opportunity of joining brilliant Neurosurgeons, Prof Krystof Bankiewicz, Prof Miroslaw Zabek, Dr Jakub Onikijuk, Dr Thomasz Pasterski and the rest of their extended surgical team in the operating theatre to observe them performing pioneering gene ‘replacement’ therapy treatment on one of our Aromatic Amino Acid Decarboxylase deficient children.
The complex and precise infusion of AAV2-hAADC to the Substantia Nigra and Ventral Tegmental Area of the
brain cannot be underestimated. The planning, the focus, the calculations, the discussion and the final
execution were ingenious! Live imaging of the initial slow infusion of the corrective aadc enzyme, encased in
a virus vector, allowed the team to be confident it was hitting the rightspot as the magical white mark
appeared on their screens.
Another of our children’s live saved!”
How to get involved...
You can donate via our #oneRAREstep JustGiving main campaign page or, if there is a particular AADCd effected child that you would like to support, please select their individual personal appeals.
Disclaimer: The AADC Trust reserves the right, in the event of us successfully raising the required funds or acquiring government funds in the country when children reside, to cover the treatment we will use any funds raised from their personal page to assist with their off treatment costs such as travel and accommodation. Any remaining funds will be used by the Trust for the purpose of this Campaign, including distributing them to other children registered for the treatment.