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NitisinoneView: Translations (EN) Original language
The AKU Society believes that Nitisinone is the best potential treatment for AKU. Our science teams have good evidence that it can treat the disease and we know of a couple of patients taking the drug that report less pain. At the moment, it is unlicensed for AKU, but in the UK, a patient can still request that their GP prescribes it. I think other countries will have different methods of getting access to treatments, but we know of a few patients in the USA and France that are also on Nitisinone.
We'd like to know of any patients taking Nitisinone - also known as NTBC or Orfadin. So please either reply here or send me a personal message. If any patients not taking Nitisinone want to know more, feel free to ask any questions.
Hi Barbara. Good to hear from you.
Nitisinone is an unproven treatment, but we believe in it enough to recommend it to patients and are attempting to get a clinical trial to prove that it works. I'll keep everyone up-to-date on our progress here. The few patients taking the drug say that they feel reduced pain and we have scientific evidence that it does reduce homogentisic acid (the black pigment that discolours your bone and urine), so we're hopeful for the future. However there is still some way to go.
We know of some patients in the UK, USA and France that have been prescribed Nitisinone off-label by their GP - I don't know how healthcare works in Italy, but it may be a good idea to talk to your doctor. Tell him he can contact me for more info.
Also, I'd suggest you get in contact with AIMAKU (the Italian AKU Society) headed by "sestinis" on here.
Thank you Oliver, I really appreciate your help! Anyway, if your clinical trial starts, it will only involve people from your country, I suppose? If not, please feel free to contact me... I will try to get in touch with AIMAKU, though.
Hope to hear from you very soon