One way of stopping the build up of these toxins is to stop one of the enzymes further up the chain. In the car analogy, it might be safer and easier to stop the production line at an earlier stage so that you don’t get a build up of cars without engines.
This is how Nitisinone works. It blocks an enzyme further up the chain called 4-Hydroxyphenylpyruate dioxygenase (HPPD). Stopping this enzyme from working prevents the production of HGA in the first place, and stops the rest of the chemical reactions from happening in the pathway. This also stops the production of toxic chemicals in HT-1 patients.
Nitisinone therefore breaks the pathway before the toxic HGA can accumulate.
For more information about nitisinone and the DevelopAKUre trial, please contact the DevelopAKUre team. Your doctor may also be able to give you more information and background about nitisinone.