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Tips, information, and resources for patients and families managing HT-1.
You know those life-changing moments that will never fade from your memory? Mine came in the form of a phone call eight days after we brought our brand-new baby daughter home from the hospital for the first time.READ MORE
Cold, blustery weather offers the perfect opportunity to huddle up inside and enjoy a hot, home cooked treat with friends and family.DIG IN
This past summer, five members of the Sobi team headed to North Carolina to volunteer at the Network of Tyrosinemia Advocates' (NOTA) first-ever camp for children with HT-1 and their families.READ MORE
When her son was diagnosed with HT-1, Robynne didn’t know anyone else who was going through what she was.WATCH VIDEO
Need recipe ideas? Help navigating insurance? Want to connect with the HT-1 community? Orfadin4U is here to support you.LEARN MORE
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.DIG IN
Managing a rare disease like HT-1 might cause feelings of isolation. We asked parents caring for children with HT-1 for their advice.READ MORE
From summer campers to college students, there is a lot to celebrate in the Orfadin Generation.CHECK IT OUT
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.DIG IN
You know those life-changing moments that will never fade from your memory? Mine came in the form of a phone call eight days after we brought our brand-new baby daughter home from the hospital for the first time.
On the other end was a nurse from our pediatrician’s office. One of Claire’s newborn screening tests had come back abnormal. My husband and I were both in shock. How could this be? Everything seemed fine when we brought her home.
We went to the doctor the next day. He told us that Claire had a metabolic disorder called hereditary tyrosinemia type 1 (HT-1). She would need to be on medication, formula, and a low-protein diet for the rest of her life.
My husband and I were devastated. So many questions flooded into my head: How did this happen? Why did this happen? Where did this come from?
I knew I had a lot to learn, but I was going to be my daughter’s advocate every step of the way.
Claire’s HT-1 affects the whole family. Because we have to plan for every possibility, we’ve lost some of our spontaneity. But we don’t really have to make sacrifices. We just always try to stay one step ahead. When we go on vacation, for instance, we drive so we can take a cooler filled with low-protein food, and a toaster oven to cook it in. When we eat out, we’re very careful about selecting restaurants that will have things that Claire can eat.
Right now, my husband and I record what she’s eaten, but someday soon, Claire will be able to do that herself. Every day, she’s becoming more independent. Some days she wants to help make her lunch. When I’m not pressed for time, I want her to be involved. One day, she’ll be the one making decisions about what to eat. She’s very good about remembering to read the labels and weigh her food. She knows the importance of taking her medicine now, and sometimes I worry about whether she’ll keep it up during her teens and college years. But I try not to get too far ahead. I try to focus on what’s right in front of us.
When Claire was first diagnosed, we felt so alone. It’s comforting to know there are other families who are faced with the same concerns we are. That’s why I’ve become so active in the HT-1 community. It’s become a huge part of our lives. I hope that by sharing our story, I can make the journey a little easier for other families living with this condition. And I look forward to the day when Claire will share her story as well. Having a supportive community that understands what you’re going through is invaluable.
It isn’t easy to have a child with HT-1, but we’ve been able to adjust by planning and staying one step ahead.
Today, although that life-changing phone call is imprinted on my memory, I have so many more happy memories with Claire and our family. Most days, Claire is a joyful, optimistic, silly kid. She doesn’t let HT-1 stand in her way. And neither do I.
Want to speak to an Orfadin Ambassador like Shannon?SIGN UP HERE
Cold, blustery weather offers the perfect opportunity to huddle up inside and enjoy a hot, home cooked treat with friends and family. Below you’ll find three protein-conscious dishes perfect for warming up on a winter’s eve.*
Have a recipe you’d like to share?
Email us at firstname.lastname@example.org.
*Every patient with hereditary tyrosinemia type 1 (HT-1) is different. Talk to your doctor about what’s appropriate for your specific medical and dietary needs.
By Trista, Vice President of Communications and Patient Advocacy at Sobi
This past summer, five members of the Sobi team headed to North Carolina to volunteer at the Network of Tyrosinemia Advocates’ (NOTA) first-ever camp for children with HT-1 and their families. Sobi was proud to be the presenting sponsor of this historic event for the HT-1 community.
On the first night, the president of NOTA asked the families gathered in the camp dining hall to raise their hand if they’ve never met another person with HT-1. About half of the hands in the room went up, illustrating the isolation that so many rare disease patients face. Now that they were finally in the company of others who shared and understood their experience, the children formed deep, fast friendships. These connections were equally important for the caregivers, who eagerly traded advice on low-protein recipes and apps for tracking phenylalanine.
Over the next two days, the 18 families at camp bonded while fishing, boating, riding horses, and playing mini-golf. Several new friends teamed up together to perform in the talent show. And many patients achieved things they never thought they would be able to do. Despite the tremors she suffers from, one patient learned to hit a bullseye in archery.
By the end of camp, the kids were all weeping and hugging — and so were many parents and even us volunteers! I feel very fortunate to work for a company that is committed to a community as vibrant and resilient as the HT-1 community.
When her son was diagnosed with HT-1, Robynne didn’t know anyone else who was going through what she was. Now, she’s giving families living with HT-1 something she didn’t have: support from someone who truly understands.
Need recipe ideas? Help navigating insurance? Want to connect with the HT-1 community? Orfadin4U is here to support you.
With Orfadin4U, you have access to:
Patient Assistance Program
A supportive HT-1 community + mentorship program
Personalized pharmacy services
HT-1 recipe ideas + lifestyle support
Insurance and co-pay assistance
“Orfadin4U works hard for us, helping my daughter get her medication. They provided pamphlets that explain tyrosinemia and how it’s treated. When my daughter started public school, I gave those out to the principal, the nurse, and her teacher, so everybody could learn.”
– Shannon, cares for daughter with HT-1
Call 877-473-3179 (between 7:00 am – 6:00 pm CST) Email email@example.com
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.
If they have a menu online, check it out to make sure the restaurant has some HT-1-friendly options. You may also want to call and ask if they’re flexible about substitutions.
Some restaurants have gluten-free menus. Ask your server if they offer one. If they don’t, look for the little asterisks or symbols next to the menu items. These are sometimes used to call out specific types of dishes, like vegan, nut-free, or gluten-free options.
The sides section of the menu usually has plenty of low-protein options, including french fries, cooked vegetables, salads. Try mixing and matching or opting for a double order.
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Managing a rare disease like HT-1 might cause feelings of isolation. We asked parents caring for children with HT-1 for their advice.
“In the beginning, I felt very isolated. My first piece of advice is to lean on your medical support team. Second, don’t be afraid to express your feelings to your family, friends, and network. The people in my network supported me and even bought me things that might help, like cookbooks.”
— La-Tasha, cares for 11-year-old daughter with HT-1
“Our clinic is very active and hosts events. We’ve had cooking classes, blueberry picking, and guest speakers. Even though my daughter is the only tyrosinemia patient there, we connect with other metabolic patients through shared dietary restrictions.”
— Shannon, cares for 7-year-old daughter with HT-1
“We felt nervous when our son was first diagnosed, not knowing what the future would look like. Empowerment has been important for us. We give him the tools he needs to make decisions, understand, and be able to explain tyrosinemia to other people, and that has made him feel more confident. It’s a learning process.”
— Amanda, cares for 8-year-old son with HT-1
Want to talk to someone who truly understands the HT-1 journey?connect with a mentor
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.DOWNLOAD THE GUIDE
Try low-protein veggie burgers with plant-based cheese
Try tacos with grilled veggies
Try BBQ cauliflower
Try pulled jackfruit with BBQ sauce
Try grilled eggplant + portobello mushrooms
Try dairy-free ice cream, sorbet, or Italian ice
Try zucchini noodle salad
Try strawberries and coconut-based yogurt
S'mores with dark chocolate, all-fruit smoothies, grilled pineapple, veggie kebabs, corn on the cob, fruit salads + veggie salads
*Every patient with HT-1 is different. Talk to your doctor about what's appropriate for your specific medical and dietary needs.
INDICATION AND USAGE
Orfadin is a synthetic reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase indicated for use as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1 (HT-1).
IMPORTANT SAFETY INFORMATION
Tyrosine levels can increase in the blood if you do not restrict tyrosine and phenylalanine in your diet while taking Orfadin. Too much tyrosine in the blood can cause serious eye problems or other complications.
Do not adjust your Orfadin dosage in order to lower the tyrosine levels in the blood.
A reduction in the number of white cells and platelets in the blood have been observed during treatment with Orfadin. Your platelet and white blood cell counts should be monitored regularly during Orfadin treatment.
The most common adverse reactions to taking Orfadin are liver cancer, liver failure, low platelets or white cells in the blood, and complaints related to the eyes, including conjunctivitis, corneal opacity, inflammation of the cornea, and extreme sensitivity to light.
Tell your physician promptly if you have unexplained eye symptoms, rash, jaundice (yellowing of the skin or whites of the eyes) or excessive bleeding.
Use Orfadin during pregnancy only if the potential benefit justifies the potential risk to the fetus.
Nursing women should discontinue either Orfadin or breast-feeding based on the recommendation of your healthcare professional.
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