How does Orfadin affect the overall survival of HT-1 patients?
Patients presenting with HT-1 under 2 months of age and treated with dietary restrictions alone had 2 and 4-year survival probabilities of 29% and 29%, respectively; HOWEVER- patients presenting with HT-1 under 2 months of age and treated with dietary restriction and nitisinone had 2 and 4-year survival probabilities of 88% and 88%, respectively in the study patients. Additionally; Patients historically presenting with HT-1 under 6 months of age and treated with dietary restriction alone had 2 and 4-year survival probabilities of 74% and 60%, respectively; HOWEVER- patients presenting with HT-1 under 6 months of age and treated with dietary restriction and ORFADIN had 2 and 4 year survival probabilities of 94% and 94% respectively in the study patients.
