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Téa: My name is Téa. This is my dad, [00:00:30] Armin. This is my mom, Michelle. This is my grandpa, Peter, and this is my dog, Lulu. I have a sister named Isla. She lives in Arizona, a long way from me. She is a very good sister and I love her to the moon and back. This is Peppermint and this is Kiwi, and that's [00:01:00] my family. I was born with Alagille syndrome, and it hurts, and it's painful and your body tells you to itch, and I'm trying to stop.

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Michelle: When I first found out that Téa had Alagille syndrome, [00:01:30] my heart broke, and I was lost. I felt like everything that I've always wanted, not that it was taken away from me, but that joy of a newborn and having a baby in your arms, it was so hard. It was hard on us. It was hard on our marriage.

Armin: I think I was in denial, and I still might be. My first instinct was, [00:02:00] “why?” We didn't know what to do.

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We were just lost. The girl that you always want, seeing her going through something that no kid should go through, so tiny, so small, it was hard.

Michelle: When we heard about her condition, they were saying, [00:02:30] “there can definitely be lots of itching.” She was 3 months old when she was diagnosed. She couldn't tell me that she was itching, so I really had to look for the signs. There was lots of itching and wiggling around. The physicians described it to me as having internal itching. It's almost like having poison ivy inside your body. For her, it was a lot of reaching for the ears, lots of scabs, lots of bleeding and same with the head. She would take both of her hands and [00:03:00] just scratch.

Armin: She couldn't sleep. She would scratch all night and you as a parent don't know what to do, how to help them. We would get up and just scratch her until she goes to sleep.

Peter: My daughter, she goes to work. She brings the baby by us. We have a crib for her, and we put them in a bed, the same house she laying down and her hands go all around in the [00:03:30] body, more time go in her legs, back, all over.

Téa: At nighttime, the itching was the worst on my ears, my legs, and my toes. That was the worst part of the night. In the morning, I would wake up and I would see that there's blood all over my sheets. I said to myself, "why am I the only one who has this?" [00:04:00] At school it was hard, during PE class I think that was the worst time of the day that I itched. Some kids at school were not being very nice, and I did not like it. I would say to the kids who were not being nice to me, I would say, "you should understand that I have Alagille syndrome and now I'm itching and I can't help it. [00:04:30] I don't know what to do."

Michelle: I categorized her itch as mild, and I think because I just made it so—it was so normal, it was just a part of our life at that point that I regret it because it wasn't normal. It was affecting her daily life. There was an opportunity for us to get into a trial with [00:05:00] LIVMARLI. Téa wasn't able to access the trial because I categorized her as mild. Hearing other people's stories on their itching, I was like, “okay well, she's not doing that. It's not that bad, or if it's cool in the home, she's not itching that bad.” I always found something to be like, “okay, it's not that bad,” but in reality it was. [00:05:30]

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Michelle: We first learned about LIVMARLI through the clinical trials, so we knew that there was something in the works for itching, which we were really excited about. Téa started LIVMARLI in February of 2022, [00:06:00] when she was 8 years old. After about 14 days on LIVMARLI, we noticed a drastic change in her itching. Her first night we knew that LIVMARLI was working for her. In the middle of the night, I didn't hear her itching, and she was snoring, and it was so funny to me, but also very sad to know that it took 8 years [00:06:30] for her to sleep all night without sitting up and itching in the middle of the night. In the morning, she ran into my room and she's like, "Mama, I slept so good."

Armin: That night was definitely one of the best nights of our life. Just hearing her snore and sleep and not itch. [00:07:00]

Michelle: When she gets home from school, she's not running to take her socks off and itch. She is itching less and sleeping through the night, which is amazing for her. She doesn't have the blood on the sheets like we did before. She's rested. She's happy.

Peter: Now, Téa is taking LIVMARLI, I feel so happy. It’s helping her. She’s not scratching. She sleeps good. [00:07:30] She's full energy.

Téa: LIVMARLI helps me sleep better because I itch less. I'm not scratching and it makes me feel better, and I'm excited that I can do more things.

Armin: Tell me when it's sauce time.

Téa: Okay.

Michelle: When Téa was prescribed LIVMARLI, I received a call from Mirum Access Plus, and it was just a rundown of the [00:08:00] medication, potential side effects, how delivery was going to work, if I had any questions or concerns, who to call, and quite frankly, it was probably the easiest start of a new medication I think I've ever been through. We received our first dose of LIVMARLI 2 weeks after I spoke to Mirum Access Plus. We went through, it was approved through insurance, and we received front door service of our prescription. [00:08:30] Our doctor did mention that there were some side effects with LIVMARLI.

Téa did experience some cramping, stomach cramping, but those have now subsided. It's better to talk about it than to hide from it, and it's good for her. It's good for us, and it's good for our family. [00:09:00] We were never ashamed, but it was almost like you're almost in denial. There's more people out there like us who are like, we don't want to talk about it. Let's just keep going. I definitely feel like the Alagille Syndrome Alliance is such a great—there's so much amazing information. Roberta is very communicative on the Alliance [00:09:30] and it's just a great resource, and it's amazing to see how many kids there really are and you're not the only one that's feeling this way.

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Armin: Advice that I would give other dads going through this, would be to be strong; do a lot of research. Be there for your kids; don't look at your kids [00:10:00] any other way, as they're not normal. They're the same as every other kid. Spend as much time you can with them.

Michelle: If I can give other caregivers advice, it would be, join the Alagille Alliance. It really makes a huge difference. Build that relationship with your physician. They're all different. Find one that suits you and your family because it really made [00:10:30] a huge difference for us. There's light at the end of the tunnel. He might not know everything, but that light just seems a little bit brighter.

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Narrator:

INDICATION

LIVMARLI is a prescription medicine used to treat cholestatic pruritus (itch) in patients with Alagille syndrome 3 months of age and older. It is not known if LIVMARLI [00:11:00] is safe and effective in children under 3 months of age or in adults 65 years and older.

IMPORTANT SAFETY INFORMATION

What are the possible side effects of LIVMARLI?

LIVMARLI can cause serious side effects, including:

Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome but may worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your health care provider should do blood tests before starting and during treatment to check your [00:11:30] liver function. Tell your health care provider right away if you get any signs or symptoms of liver problems, including:

• nausea or vomiting
• skin or the white part of the eye turns yellow
• dark or brown urine
• pain on the right side of the stomach (abdomen)
• loss of appetite

Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment. Tell your health care provider right away if you have any of these symptoms more often or more severely than [00:12:00] normal for you.

A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamins A, D, E, and K) stored in body fat. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment.

Other common side effects reported during treatment are gastrointestinal bleeding and bone fractures.

Tell your health care provider about all medicines that you take. LIVMARLI may affect [00:12:30] the way some other medicines work, and some other medicines may affect the way LIVMARLI works. If you take a medicine that lowers cholesterol by binding bile acids, such as cholestyramine, colesevelam, or colestipol, take it at least 4 hours before or 4 hours after you take LIVMARLI.

LIVMARLI is taken by mouth, 1 time each day, 30 minutes before a meal in the morning. Be sure to use the provided oral dosing dispenser to accurately measure the dose of medicine.

These are not all of the possible side effects of LIVMARLI. [00:13:00] Call your health care provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

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