On Wednesday 24th of July, the AKU Society will be hosting an online webinar with RareConnect (#). The webinar will be about our second clinical trial, SONIA 2, which will test the effectiveness of nitisinone as a treatment for AKU. SONIA 2 is expected to begin later this year and will run over .. ...more
What is orphan designation? Rare diseases are often referred to as orphan diseases because patients may feel orphaned from the rest of society. Orphan drugs are drugs which are used to treat rare diseases. Drugs are designated to treat orphan diseases if they meet three main criteria. Fi.. ...more
Alkaptonuria (AKU) is a genetic disease which damages the bones and cartilage, causes severe pain and leads to health problems such as osteoarthritis, heart disease and kidney infections. It is extremely rare and generally affects only one in every 250.000 people worldwide. AKU prevents the body.. ...more
Last week, we headed to Innsbruck, Austria to attend the Society for the Study of Inborn Errors of etabolism's annual symposium. Inborn errors of metabolism are rare genetic conditions where the body cannot completely break down food. Alkaptonuria (AKU), was the very first inborn error of metabo.. ...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