Our ‘mother’ society in the UK has just launched a crowdfunding campaign to raise funds for a global Black Bone Disease registry in order to gather information about AKU patients around the world in one single place.
This will allow all of the AKU societies representing patients throughout the world to make the case for access to the life-changing drug nitisinone in the many countries where it is not yet available or is available, but not affordable.
This effort will also help us prepare plans for gene therapy and other new treatment studies.
You can donate here: https://chuffed.org/project/theakusociety
Let us know if you have any questions about how this affects patients, families, physicians, and researchers in North America. firstname.lastname@example.org