The AKU Society successfully led the 10 year development programme of the drug nitisinone for the ultra rare genetic disease Alkaptonuria (AKU, or Black Bone Disease). But we are now learning that the disease is even more serious than originally thought and that limiting the drug’s side effects requires a strict low protein diet.
That’s why the AKU Society has launched its own drug discovery and development programme that it is funding at the University of Liverpool in order to develop new drugs that will resolve these issues. Find out at our talk what challenges face patient groups carrying out their own drug discovery programme and how to overcome these.
Nick Sireau, PhD, Chair and CEO, AKU Society