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Terisa: My name is Terisa. I have three boys. The oldest, Brian, is 14, Tyler is 10, and Benny is 5. [00:00:30] I think that being a mom is probably what my calling's been. I love being their mom. I love being just involved with raising them. It's what I've always wanted to do. I've always wanted to have kids. I didn't really know what I wanted to do with work. I just knew I wanted my job to be being a mom. The most rewarding thing is just watching them grow up, just seeing their different personalities. They're so unique.
They all have their own character traits, their own interests. Tyler's hobbies are swimming, [00:01:00] playing basketball, throwing the ball around with his brothers. He likes to do that. He likes to cook with me. We like to just hang out and watch movies and videos. He loves video games. That's his favorite thing right there. When I was pregnant with Tyler, they did a very thorough check, the 18-week fetal survey, just to be proactive since I was born with a heart condition. That's when they found the hole in his heart and some issues. They diagnosed him prenatally [00:01:30] with tetralogy of Fallot.
He was born at 39 weeks. Everything looked good. They took an X-ray of his heart. That's when I saw the butterfly vertebrae, which is now knowing that that's the first marker that we found for Alagille syndrome. Then he went home, and we were looking forward to surgery day. That was it. He started getting really jaundiced, even though he was eating really well. The pediatrician had just run some basic bilirubin tests. He was [00:02:00] really high, but he didn't think it was dangerously high to send him to the hospitals.
We were going back and forth between cardiology for a week and then pediatrician for a week just to make sure his heart was stable until he was ready for surgery. In the morning of pre-op, they just threw on a liver function panel test just to see if the bilirubin dropped. Next thing you know, surgery's canceled, and he's whisked upstairs for a liver ultrasound, and then that's where they were able to diagnose the bile duct paucity. [00:02:30] They went right from there and clinically diagnosed him with Alagille syndrome.
When he was diagnosed with Alagille syndrome, I just remember standing there and everything spinning [00:03:00] around me. I just felt like I was in this tornado of like, what? This just happened to me? I just did everything you told me to do, and now I have this diagnosis. The light in the tunnel there was finding the Alagille support page and finding people that were going through exactly what I was going through, and being able to also connect with families that had the same age child as my kid. It was great to see adults [00:03:30] and say, "Wow, these kids have a future."
Ironically, it felt very comforting to have someone on my side. Learning about bile acids through the Alagille support page, I realized that that was a big factor to his itch. Basically, your liver has no natural flow of the bile, so it just reabsorbs, and it's going through your bloodstream. Everywhere your blood is from head to toe [00:04:00] is going to be itchy. I started noticing him rubbing his eyes a ton. It was more than just the hunger cues, more than the tired cues. We went back into another pediatrician and asked, "What's going on?" They said, "No, it's not allergies. He's got to go through the whole season before it's diagnosed with allergies." It was just brushed off as, "We don't know what that is." The itch was getting worse. Every time we did a diaper change, he was scratching at his legs or his diaper area and then [00:04:30] rubbing the eyes constantly. That was the big thing for him. Then it moved into putting toddler socks on his hands so that they went all the way up to his arms. That way, at night, he wasn't digging into his ears and making them bleed everywhere.
The itch normalized for us. We just knew it was there, but at the same time, we really discredited a lot of the itch and a lot of the liver involvement to just being mild, even though we later learned that it was [00:05:00] much more severe than mild. It just was changing bed sheets consistently, bandaging up all the cuts. Unfortunately, grieving the fact that I couldn't do the bed sharing and the cuddling with Tyler because he sweat so much. Then it made him more itchy. Then, trying to sleep with him, he was tossing and turning and rolling and scratching.
I was like, "I can't do this. I'm not sleeping. He's not sleeping." Naps were few and far between. He just was never settled for a nap time because he was so itchy. There was no [00:05:30] medication out there at the time for Tyler to take that actually worked for him to relieve that itch.
Tyler: The itch is just bad. It's horrible, and it keeps me from doing stuff. The itch just feels like an itch, like a bug bite or something. Then you go, and it goes from here to your back, and it goes all over, and then you can't stop.
Terisa: [00:06:00] We had learned that LIVMARLI was getting close to being FDA approved, and we were very excited about something being an option for him again. We worked with our doctor and [00:06:30] got him on LIVMARLI. Tyler was a good candidate for LIVMARLI because he had Alagille syndrome, but then he had that very persistent pruritus. When he turned 10, he started LIVMARLI, and that was great. It was a really fun day for him because it was his birthday, but also it was the start of what could be his new normal, not itching as much as he had been.
On his 10th day, was when we started realizing he's sleeping through the night. He's not tossing and turning. [00:07:00] There had been no new scabs, no bandages on his arms and legs. That was a sight to see, to not have the bandages so much, but then also just to see him sleeping and his brothers sleeping because they all shared a room, and they weren't being woken up by him tossing and turning so much. Once his doctor prescribed LIVMARLI, we got connected with the Mirum Access Plus program, and they got it right on our doorstep in less than two months.
It's been nice to not have to be chasing down doctors [00:07:30] for refills and going to the pharmacy to pick things up. It's just so convenient having it right to our door. One side effect that we've been battling is of GI cramping. That's just something that we knew it was a possibility of this happening. We had to work with our doctor to make sure that we could manage this for him, and let it be so that he could still attend school and do his daily activities. [00:08:00] I think he's great now. We're going strong on 10 months. What do you think?
Tyler: I think we're doing good.
Terisa: You feel good on the medicine?
Tyler: Yes.
Terisa: I know 10 years is a long time, but I'm glad he wasn't 20, and he just got his hands on this. I wish it could have been sooner for him, but I'll take 10 years and then the rest of his life.
Tyler: Having Alagille syndrome doesn't really make me feel much [00:08:30] different. It just makes me feel like myself because it doesn't affect what's on the outside. It mostly affects what's on the inside. Now that I'm on LIVMARLI, I'm itching less, and I'm sleeping better.
Terisa: There's people out there that are willing to connect and happy to connect and make sure that you feel supported through your journey, because in the beginning, it can be very overwhelming, and you can feel like you're drowning. There's light on the other side, and there's [00:09:00] possibility of having happiness as part of your everyday.
Narrator:
INDICATION
LIVMARLI (maralixibat) oral solution and tablets are a prescription medicine used to treat cholestatic pruritus (itch) in patients who are 3 months of age and older with Alagille syndrome.
It is not known if LIVMARLI is safe and effective in children with Alagille syndrome who are under 3 months of age. It is not known if LIVMARLI is safe and effective in adults [00:09:30] who are 65 years of age and older.
IMPORTANT SAFETY INFORMATION
What are the possible side effects of LIVMARLI (maralixibat) oral solution and tablets?
LIVMARLI can cause serious side effects, including:
Liver injury. Changes in certain liver tests are common in patients 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 and physical exams before starting and during treatment to check your liver function. [00:10:00] Tell your health care provider right away if you get any signs or symptoms of liver problems, including:
- nausea or vomiting
- your skin or the white part of your eye turns yellow
- dark or brown urine
- pain on the right side of your stomach (abdomen)
- fullness, bloating, or fluid in your stomach area (ascites)
- loss of appetite
- bleeding or bruising more easily than normal, including vomiting blood
Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain during treatment. Diarrhea can also cause the loss of too much body fluid (severe dehydration). Your health care provider may advise you to monitor for new or worsening stomach problems, including stomach pain, diarrhea, blood in your stool, or vomiting. Tell your health care provider right away if you have any new or worsening signs or symptoms of stomach and intestinal problems, including:
- diarrhea
- more frequent bowel movements than usual
- stools that are black, tarry, or sticky, or have blood or mucous
- severe stomach-area pain or tenderness
- vomiting
- urinating less often than usual
- dizziness
- headache
- A condition called Fat-Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body 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, and may monitor for bone fractures and bleeding, which are common [00:11:30] side effects.
Tell your health care provider about all medicines that you take, as LIVMARLI may interact with other medicines. If you take a medicine that lowers cholesterol by binding bile acids, such as cholestyramine, colesevelam, or colestipol, take LIVMARLI at least 4 hours before or 4 hours after you take that medicine.
Your health care provider may change your dose or temporarily or permanently stop treatment if you have certain side effects.
LIVMARLI is available in oral solution and tablet formulations. LIVMARLI [00:12:00] is taken by mouth, 1 time each day, 30 minutes before a meal in the morning. For the oral solution, 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. For more information, ask your health care provider or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.
Please see full Prescribing Information, including Patient Information and Instructions for Use, for LIVMARLI at LIVMARLI.com.
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