Julee: [00:00:00] So what was it like watching your child grow up with the itch with the Alagille syndrome?
Lisa: It was very challenging. From the time she was able to scratch, she did. She would scratch her ears [00:00:30] until they bled, and we had no idea what was going on. The docs, they couldn't tell us anything. They had no idea. Until we got her diagnosed at 11 months, we had no idea if she was going to live. She was itching very bad. They really couldn't do anything. They had already put her on 2 or 3 medications, and none of those helped, but we managed it the best way we could.
Julee: Yes.
Lisa: How about you? [00:01:00]
Julee: She was constantly itchy and fussy. There was nothing I could do to console her. She'd itch her ears and her feet, and it was nonstop. It was just painful to see her suffering so much. Then as a mother, we feel like we're responsible for stopping that, and [00:01:30] I couldn't stop that with her. Kids made fun of her sometimes, and I think that was probably the most heartbreaking thing. As a mother, the other kids didn't understand what was happening, and it made me very angry at different times.
Lisa: What changes did you notice in Emma once she started taking LIVMARLI?
Julee: Instead of seeing her constantly itching, she was able to use those fingers to create things, [00:02:00] to play instruments. I feel like I got to see her blossom. Her self-confidence took off. She wasn't worried what other people were thinking about her. It was beautiful.
Lisa: Abby as well, after she started taking LIVMARLI, she said she wasn't waking up itching as much. She's just feeling much better after LIVMARLI.
Julee: Now that Abby's a little bit older, what ways is [00:02:30] she taking charge of her own health?
Lisa: She's in charge of taking her LIVMARLI, making sure that she gets it, and making sure she stays on her routines, and seeing her doctor, keeping her appointments, doing all those things that we used to do when she was younger. When she first went to college, because I worried a little bit that she wouldn't take care of herself. She takes her medication, and [00:03:00] she stays on top of her doctor's appointments, and I'm very proud of her. I'm very proud of her.
Julee: That's nice.
Lisa: I'm very proud of her.
[theme music]
Lisa: What was your process of getting LIVMARLI?
Julee: It was surprisingly very easy. They contact you. They communicate every step [00:03:30] of the way. Then they plan when to send LIVMARLI out to you. I think what I really liked most was they helped me with the insurance part of it. Because to me, that was always the most difficult part. What was your experience like?
Abby: Almost exactly the same. I'm on my own insurance, and they were really helpful every step of the way. Every time I would call, they would call me back as soon as they said they were going to.
Lisa: They [00:04:00] communicate really well, don't you think?
Emma: Yes.
Abby: Yes. I love my navigator. She's amazing. She is sweet, awesome, calls when she says she's going to. At the beginning, it was like every week or every 2 weeks. She just wanted to follow up on how LIVMARLI was going. Now it's like every 3 months, and she calls right on the dot, and it's nice to just have someone that I know that I can call if I have any questions at all.
Julee: I've always known what was inside of Emma [00:04:30] and what her potential was, but with all the itching, it was really hard for Emma to be the person that she could be because the itching controlled her life. She reminds me of a little butterfly that has taken off and blossomed.
Lisa: I don't know. No parent really knows what it's like until they [00:05:00] have a sick child. There was a time that I didn't know if we'd ever find anything that would help with the itching, but I feel like that we've found something now that is helping you, and I am not as afraid. I'm not as worried all the time. I'm very proud of her and her beautiful life that she's building. I'm thankful for LIVMARLI, y'all, really.
Speaker:
INDICATION [00:05:30]
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 who are 65 years of age and older.
IMPORTANT SAFETY INFORMATION
What are the possible side effects of LIVMARLI (maralixibat) oral solution and [00:06:00] 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. 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 [00:06:30] 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 [00:07:00] 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 [00:07:30] 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 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, [00:08:00] 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 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 [00:08:30] 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. [00:09:00]
[00:09:07] [END OF AUDIO]
