Our ultimate goal is to cure AKU. We are committed to funding and collaborating on research into increasing our understanding of AKU and working on potential treatments and cures for the condition.
Our current projects include treating nitisinone-induced hypertyrosinaemia, gene therapy, mRNA therapy, and our global AKU registry.
DevelopAKUre
The DevelopAKUre programme was a series of major international clinical trials, run by a consortium...
AKU Registry
Our global patient registry collects data on the progression and prevalence of AKU across the world...
Funding Opportunities
We are constantly looking to fund research into developing new treatments and potential cures for AKU.
Hypertyrosinaemia
We are currently conducting research into treating nitisinone-induced hypertyrosinaemia ...
Research & Funding Policies
As a patient-group charity, we are committed to ensuring the medical research we fund is always conducted to meet...