orfadin patient assistance program

Orfadin Patient Assistance Program, a patient assistance program provided by Rare Disease Therapeutics, Inc, offers a year’s supply of Orfadin at no cost to those who are eligible for the program. Eligibility is based off of the following requirements: - You must not be covered by private or public health insurance.

Full Answer

How much does Orfadin cost?

The cost for Orfadin oral capsule 2 mg is around $5,748 for a supply of 60 capsules, depending on the pharmacy you visit. Prices are for cash paying customers only and are not valid with insurance plans.

What is HT 1?

Background: Type 1 hereditary tyrosinaemia (HT1) is a rare metabolic disorder caused by an enzymatic defect in the metabolism of the amino acid tyrosine. Primary treatment for HT1 is nitisinone (Orfadin) in conjunction with a low-tyrosine/phenylalanine diet.

What is nitisinone used for?

Nitisinone is used to treat hereditary tyrosinemia type 1, which is too much tyrosine in the blood. It helps to prevent high levels of tyrosine in the blood. Treatment with nitisinone and diet changes may slow the progression of the disease, but will not cure it.

Which disorder is caused due to deficiency of enzyme tyrosinase?

Tyrosinemia type II is an autosomal recessive genetic disorder caused by a deficiency of the enzyme tyrosine aminotransferase. Affected children do not have liver abnormalities.

How do you test for tyrosinemia?

Tyrosinemia is diagnosed based on blood tests and urine tests. In both the acute and chronic forms of the disease, liver function tests are often abnormal. Low serum albumin and clotting factors are also frequently found.

Is nitisinone a drug?

Nitisinone works by helping to prevent the formation and build-up of several toxic substances that cause damage to the liver, kidneys, and nervous system. This drug must be used along with a diet low in protein, tyrosine, and phenylalanine.

What does high tyrosine mean?

Tyrosinemia is a genetic disorder characterized by disruptions in the multistep process that breaks down the amino acid tyrosine, a building block of most proteins. If untreated, tyrosine and its byproducts build up in tissues and organs, which can lead to serious health problems.

Why do patients with Tyrosinemia benefit from the drug nitisinone NTBC?

It blocks the catabolic pathway of tyrosine, thereby leading to a reduction in the accumulation of toxic metabolites in HT-1.

What current research is being done to develop a cure for albinism?

A small pilot clinical study at the National Eye Institute (NEI) suggests that the drug nitisinone increases melanin production in some people with oculocutaneous albinism type 1B (OCA-1B), a rare genetic disease that causes pale skin and hair and poor vision.

What is tyrosine metabolism?

Tyrosine is an aromatic amino acid important in the synthesis of thyroid hormones, catecholamines, and melanin. Impaired catabolism of tyrosine is a feature of several acquired and genetic disorders that may result in elevated plasma tyrosine concentrations [1]. TYROSINE METABOLISM.

What is NTBC in Tyrosinemia?

Nitisinone (NTBC) is a drug used to prevent the production of these toxic metabolites, by blocking the catabolic pathway of tyrosine at an earlier stage (4-hydroxyphenylpyruvate dioxygenase) (Fig.

One step ahead

All individuals featured in this website have experience with Orfadin but may no longer be on treatment.

Cozy cooking

Cold, blustery weather offers the perfect opportunity to huddle up inside and enjoy a hot, home cooked treat with friends and family.

Orfadin Ambassadors: Robynne

When her son was diagnosed with HT-1, Robynne didn’t know anyone else who was going through what she was.

Dining out: quick tips

Ordering at a restaurant can be tricky when you or a loved one has a dietary restriction. But that doesn’t mean you should miss out! Here are a few tips for families managing HT-1.

DO YOU EVER FEEL ISOLATED?

Managing a rare disease like HT-1 might cause feelings of isolation. We asked parents caring for children with HT-1 for their advice.

ALL ABOUT THE ORFADIN GENERATION

From summer campers to college students, there is a lot to celebrate in the Orfadin Generation.

SUMMER SUBSTITUTES

With all the picnics, barbecues, and ice cream trucks, summer fun can seem hard to navigate with HT-1. So we put together a handy substitutions guide to help you enjoy all the festivities the warm weather brings.

How much does Orfadin cost?

The cost for Orfadin oral capsule 2 mg is around $5,699 for a supply of 60 capsules, depending on the pharmacy you visit. Prices are for cash paying customers only and are not valid with insurance plans.

How much does a free drug card save?

The free Drugs.com Discount Card works like a coupon and can save you up to 80% or more off the cost of prescription medicines, over-the-counter drugs and pet prescriptions.

Why is oral capsule important?

Oral Capsule. Important: When there is a range of pricing, consumers should normally expect to pay the lower price. However, due to stock shortages and other unknown variables we cannot provide any guarantee. Important: When there is a range of pricing, consumers should normally expect to pay the lower price.