Medical therapy for hereditary hepatorenal tyrosinemia (hereditary tyrosinemia type 1, HT-1) with nitisinone was discovered incidentally, and is a by-product of agrochemistry.
Background: Tyrosinemia type 1 (TYR1) is a rare autosomal recessive disorder of amino acid metabolism that is fatal without treatment.
Introduction: Tyrosinemia Type 1 (HT1) is an autosomal recessive disorder caused by a defect in the enzyme fumarylacetoacetate hydroxylase in the tyrosine pathway.
Hereditary tyrosinemia type 1 (HT-1) Nitisinone is indicated for the treatment of adult and paediatric (in any age range) patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. Alkaptonuria (AKU) Nitisinone is indicated for the treatment of adult patients with alkaptonuria (AKU).
The FDA has approved Orfadin (nitisinone) 20 mg Capsule, from Swedish Orphan Biovitrum, for the treatment of Hereditary Tyrosinemia Type...
Hereditary tyrosinemia type 1 (HT-1) Orfadin is indicated for the treatment of adult and paediatric (in any age range) patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. Alkaptonuria (AKU) Orfadin is indicated for the treatment of adult patients with alkaptonuria (AKU).