Expanding
our knowledge
by working

with patients

At Mirum, we’re not just working for people with rare liver disease—we’re working with them, committed to doing what we can to improve their lives. We are immersed in the community, connecting with advocates and listening to patients and their families to truly understand the burdens they face. These insights lend the perspective needed to help improve and extend their lives in meaningful ways.

The truth is, we’re never short of being amazed at how remarkable these patients and families can be.

Here are just some of their stories:
Trey
Patient with progressive familial intrahepatic cholestasis (PFIC)
When Trey was younger, the main symptom we saw was itching. It would keep him up at night and he couldn’t sleep. He would itch so bad that he would scab and bleed from his ears, his legs, and his arms. We resorted to mittens on his hands when we put him to bed just so he wouldn’t hurt himself from scratching.
—John, Trey’s Dad
Cedar
Patient with PFIC
They started talking to us about being on medicine. She’ll end up itching. She’s going to have surgery. She’ll probably need a liver transplant at some point. It’s just so overwhelming. You hear all of these things and it’s not like, ‘Here is your diagnosis and here is your cure.’ It’s ‘Here is your diagnosis and here’s what she’s going to deal with for the rest of her life.’ It’s really hard to take in the weight of that at that moment.
—Emily, Cedar’s Mom
Jocelyn
Patient with Alagille
syndrome (ALGS)
I still remember the day that we got the call and we were told that she had Alagille syndrome. It was confirmed through genetics, which she had done when she had a liver biopsy and her cholangiogram. Although itching can take her mind off the things she enjoys, she is a happy girl. She loves to sing, dance, play, and interact with other children.
—Melissa, Jocelyn’s Mom

If you’d like to learn more about the above
conditions or other rare cholestatic liver
diseases, check out our resources.

SEE AVAILABLE RESOURCES