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Dani: Really careful.

Jackson: You can try it by yourself.

Dani: Good job. You got to do one at a time.

Jay: My name's Jay.

Dani: I'm Dani. We have two beautiful boys: Jackson, who's four, and Finley, who's one.

Jay: Jackson's just [00:00:30] a mover and a shaker. He is a ball of energy. He's just always on the move, but he's creative, he's energetic.

Dani: Finley's personality is a lot more mellow. He really enjoys reading books together, listening to music, and he really likes to see how things work. Jackson's pregnancy, super easy. No complications, no NICU stay, no hospital stay. It was a really straightforward pregnancy. Not with Finn [00:01:00] though. Not with Finn. Good job, Finley.

Jackson: [unintelligible 00:01:05].

Dani: We were very excited to add another little baby to our family. So we got pregnant with Finn. Something that was supposed to be joyous ended up turning into not that.

Jay: In that 20-week ultrasound where they look at all of the anatomy of the fetus, they couldn't see one of the chambers of his heart. That was just the start of what was going [00:01:30] on. So we went back for another ultrasound because he was measuring small. He was measuring, at that time, in the 11th percentile. We went back and he was measuring in the ninth percentile and that's when they found what's called an echogenic bowel. We were referred to see a high-risk pregnancy doctor every couple weeks.

Dani: Finley was born a month early, so he spent the first 2 weeks of his life in the NICU. His liver numbers were really high, but they weren't [00:02:00] super concerned yet. They were just waiting to see if those liver numbers started to track down. That's when we heard the term “bronze baby syndrome.” This is what they call when they knew that something was wrong with Finley's liver. There's this one particular photo of Jay and Finley. He was holding him up over his shoulder.

Jay: You can see the fear in that picture and that's one that will forever stick out to me, is one moment where [00:02:30] it set in that like, "Okay, we're not going home right away. When we do go home, it's not going to be what we thought it was. It's not going to be what it was like when we had Jackson."

Dani: We weren't out of the woods in this one. We knew, at this time, there was something wrong. He was still jaundiced, he wasn't gaining weight, and it was a very isolating experience. It was during COVID. I, of course, refused to leave my newborn son, so I spent a lot [00:03:00] of my time there.

Jay: It was a lot. It was hard, a lot to process.

Dani: They did a lot of different tests. They did echo outside and that's when they discovered that he had pulmonary stenosis. They wanted to do a liver biopsy to rule out biliary atresia, and really look at the bile ducts and see how they were growing. They kind of [00:03:30] told us, "We don't really have an answer," but that's when we really first heard of Alagille syndrome and that it might be something that Finley has. [00:04:00]

I'm supposed to protect him, and I feel like the mutation is my fault, even though, logically, I know it's not. It was a brand new mutation; it doesn't run in either of our families, but I couldn't do my job as a mother to make sure he was born healthy. That's why I do a lot of research into what Alagille syndrome is, what other families go through. [00:04:30]

Jay: Some of the symptoms that we were experiencing after diagnosis were the continued poor weight gain and being undersized, jaundice levels in his skin. We were starting to see signs of him itching.

Dani: The itch was one of the bigger pieces in that early diagnosis that we missed then, but now we understand it because a lot of the signs that our doctor told us to look out for: the rubbing of the face on your shoulder if you're holding him up [00:05:00] here, rubbing of the feet or belly, when they're trying to do tummy time. Then the scratching of the eyes or grabbing at the ears, all those are normal hunger cues, sleepy cues. Really trying to figure out what's the difference between the two. Looking back at it now, we definitely see that most of the signs were itching and not necessarily related to typical newborn behavior.

Finley, in the evening, his itch would be pretty bad [00:05:30]. He was waking up every 2 to 3 hours, well before that 6-hour mark that our doctor told us that hydroxyzine would last. The itch impacted Finley's life. That period of time is a blur from how exhausted we all were. Jackson slept through the night, so it didn't seem to disrupt him. But it did disrupt our lives though when we were awake because Jackson wanted to play, and Finley was really tired. Mommy and daddy were really tired. It was a pretty rough, dark time. [00:06:00]

Jackson: Do you want to try? Do you want to try and [unintelligible 00:06:04]? [unintelligible 00:06:06]. Oh, look at that.

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Jackson: [unintelligible 00:06:12] he's going to do it. He's going to do it. No. [unintelligible 00:06:15] try it by yourself. You can try it by yourself.

Jay: Our doctor had introduced LIVMARLI as a possible option for Finley pretty early on after diagnosis. They work with a number of families in the area that have kids with Alagille, [00:06:30] and they've just seen it really help with the itch in those children.

Dani: For me, why I wanted to get Finley on LIVMARLI was the itch. Seeing it wake him up in the middle of the night, seeing him try to play with Jackson, and eat his food, and do all those things, and just to see those little hands grabbing at the eyes, grabbing at the ankles and feet. Just seeing how much itching was really impacting him.

Jay: Finley started on LIVMARLI shortly after his first birthday, [00:07:00] when we had our follow-up with his GI doctor. Around that 4-week mark, we were really happy to see that he was reaching for his legs and feet less than what we would see when he would itch. He just had more time to be Finley and it's really awesome.

Dani: It was nice to see him be able to focus on something else outside the itch. We want him to thrive, be a child who isn't impacted by the itch through liver disease. We just want him to be a normal 1-year-old child. Our doctors shared that there could be [00:07:30] some side effects for Finley after taking LIVMARLI, but we didn't really see any of those. We didn't see the diarrhea, no vomiting. We didn't really see any...

Jay: ...changes in his vitamin levels. All remained stable.

Dani: None of the things that our doctors did share that we could potentially see on LIVMARLI, we didn't experience anything like that.

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Jay: I think we've really tried to just see how much [00:08:00] has changed in the time he's been on it with the itch subsiding and going down that it just made doing these things more enjoyable for him. We get to celebrate those moments and look at how much fun he's having without seeing him grabbing at his knees, grabbing at his ears like we used to see. It's hard to quantify it, but you just see him being a kid and having fun with his brother, and doing the slide and playing at the park. It just warms your heart, makes me happy. It makes us smile.

Dani: Just because he has been [00:08:30] dealt a certain hand in life, that doesn't mean his rare disease defines it. Finley is smart. He's happy and he's healthy, or stable in his own way.

Jay: The ultimate goal would be for people to just understand that Finley is just like any other kid and that he may have some differences that make him look a little smaller and look a little different, but he's still capable of doing what anybody else is. [00:09:00] We want to be a voice for Finley and other kids with Alagille. We want people to understand.

Dani: I feel like you might not use the word fortunate a lot in the rare diseases world, but we are really fortunate to have Alagille Alliance. Not too many rare diseases have this group of folks together that help provide the scientific research, help give you information and connect with other families who are also going through Alagille with you. We feel very fortunate that we're part of such a [00:09:30] well-known and...

Jay: ...tight community.

Dani: Yes.

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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 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? [00:10:00]

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 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 [00:10:30]
• 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 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 [00:11:00] fat. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your health care provider should do blood tests before starting and during treatment.

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

Tell your health care provider about all medicines that you take. LIVMARLI may affect 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 [00:11:30] 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. 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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