The International Rare Disease Research Consortium, or IRDiRC for short, launched in April 2011. IRDiRC was set up by the European Commission and the National Institutes of Health in the USA to bring together rare disease researchers and funders. IRDiRC aims to achieve two goals by 2020:.. ...more
Entry into the DevelopAKUre Blog Quiz (#) is now closed. I am pleased to announce our winner got all 10 questions correct! We will be in touch with you soon to send out your prizes. The answers to the 10 questions are: Q: If you are pregnant, you cannot take part in SONIA 2. This is part of.. ...more
Since the DevelopAKUre project began, we’ve talked a lot about our two clinical trials, SONIA 1 and SONIA 2. Some of you may remember us occasionally mentioning a third study called SOFIA. What is SOFIA? SOFIA stands for Subclinical Ochronotic Features In Alkaptonuria. Ochronosi.. ...more
DevelopAKUre is a series of major international clinical trials, run by a consortium of 12 European partners. It aims to study a potential new drug, called nitisinone, and assess its potential effectiveness in treating the rare disease, alkaptonuria (AKU).
DevelopAKUre is co-funded by a grant from the European Commission. This website is run by a UK patient group, the AKU Society. Learn more about AKU on the AKU Society's What is AKU page.
In today’s blog Chief investigator Prof. Ranganath reinforces the need for SONIA 2 pat.. ...more