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1. Orfadin (Nitisinone ) for the treatment of hereditary tyrosinemia type 1 (HT-1 ) What is HT1 ? Tyrosinemia type ( HT-1 ) is caused when the body is unable to break down tyrosine which is one of the protein’s core building block . When the tyrosine is unable to break down ; toxic substances are formed within blood known as succinylacetone . This clearly shows that if Tyrosinemia isn’t treated , it can cause liver and kidney damage and other neurological issues . How many people are affected by Tyrosinemia & is it inherited from both the parents? Tyrosinemia affects 1 in 100,000 people and must be inherited from both the parents. Does a specific gender is more prone to have HT-1 ? Both Male and Female are equally prone to having HT- 1 . Are people affected by Tyrosinemia never eat proteins ? No that’s not true though low – protein diet is required in such cases to cause build up of too much protein in child’s body but they still small amount of protein and amino acids are required in the body . Does HT-1 can be cured permanently ? No not necessarily , those who have HT-1 will likely to manage for entire life . What is Nitisone ? It is first drug which has been approved for the treatment of HT-1 Does the dose of Orfadin (Nitisinone )remains same throughout the life ? The dose of Orfadin depends on the body weight changes of a body , this is a

2. reason blood tests become necessary to quantify the levels the tyrosine in the blood Are there any other versions of Nitisinone available other than Orfadin ? Yes , Nobel’s Pharma also manufacturer Nitisinone known as Tisinon which is available in strength’s of 5mg and 10 mg which comes in a pack of 60 capsules . In what Dosage forms and strengths does Orfadin comes ? Orfadin comes in capsule form of 2mg , 5 mg , 10 mg and 20 mg . Apart from that it also comes in oral suspension 4mg/ml . Can Nitisinone be procured by the patients if not available or approved in one’s own country ? Yes , Indian pharma network Named Patient program help’s patient to procure the medicines which are not available or approved in one’s own country. Indian pharma network provides access to both Orfadin (nitisinone) which is marketed by the company Swedish Orphan Biovitrum International AB, Sweden and Tisinon (nitisinone)by Nobel Pharma which is a Turkish brand and manufactures drugs for unmet medical needs . Source - https://helpforheallth.blogspot.com/2022/09/treatment-of-hereditary-tyrosinemia.html