Contract award notice: Pnsp 65/2017 - acquisition of medicines: Nitisinone
, Sodium phenylbutyrate, Anakinra and efmoroctocog alfa
This reduced dosing frequency is approved for use in patients five years of age and older who have undetectable serum and urine succinylacetone concentrations after a minimum of four weeks on a stable dosage of nitisinone
sup] proposed that nitisinone
(NTCB), a triketone herbicide that inhibits 4-hydroxyphenylpyruvate dioxygenase by reversible rapid, avid binding (50% inhibitory concentration, 40 nmol/L), should be a therapeutic step.
The current treatments available for TT1 patients are pharmacotherapy with nitisinone
(NTBC) and/or a liver transplant.
In a pioneering trial at Royal Liverpool Hospital, patients with alkaptonuria (AKU) - known as black bone disease - are being given a drug called nitisinone
(A drug) inhibits 4-hydroxy phenylpyruvic acid dioxygenase as will be discussed in treatment options.
The treatment includes a low-protein diet with medical food or formula, as well as the medication nitisinone
23] having noted that nitisinone
(used in the treatment of hereditary tyrosinemia type 1) caused an increase in serum tyrosine levels, treated OCA1B mice with the drug and noted an improvement in pigmentation of the mice.
Unlicensed drug nitisinone
has had success in relieving symptoms during trials.
And now, after his long and painstaking research, he told the Mirror: "We've found an amazing treatment, a drug called nitisinone
A drug called nitisinone
has also been mentioned as a potential treatment for AKU; however, the long-term clinical efficacy and safety of this drug have also not been demonstrated.
He eventually found an advert for a trial in Washington to test the impact of an existing drug, nitisinone