The AADC Trust is looking for a visionaries, those with an imagination who would appreciate ground-breaking translational medicine. Those who would welcome partaking in paving a brighter future for children suffering with Ultra-RARE diseases. Brilliant, innovative people, who could imagine a sick child being medicated, with a ‘one-time’ treatment, transforming their severely disabled lives, whilst at the same time enhancing their entire family dynamic.
We are reaching out to big hearted, charitable, forward thinking investors, who would give willingly, in the knowledge they will be financially contributing to global medical change and personally affecting how we treat children suffering Ultra-RARE diseases, such as, Aromatic Amino Acid Decarboxylase deficiency (AADCd). Your investment may not return a financial profit but will deliver you something far superior and of greater benefit to humanity. Your contribution could help change the course of how we treat many ultra RARE neurometabolic diseases effected by a single gene defect, thus literally saving lives.
Your help could literally be the difference between a life or death outcome for a child suffering with AADCd.
1 in 55 million children in the world
AADCd is misdiagnosed as Cerebal Palsy,
seizure disorders and Myasthema Gravis
biggest recorded loss of AADCd children:
from AADCd each year.
mutations have been discovered on the
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!”
If you are a philanthropist interested in investing in our AADCd children and our Charity mission , please contact us and we will send you an official information pack.
Contact: Charity Manager
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, that in the event it successfully raises the required funds or acquires government funds from the country where a child resides, to cover the treatment we will use any surplus 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.