Making a Positive Impact on the Lives

of Patients With Rare
Diseases

We are committed to developing safe and effective therapies for people with rare diseases. Ultimately, we want to help patients and their families experience a bright future.

Supporting the community through remarkable science

Mirum is committed to putting the needs of patients first. We’re transforming lives devastated by rare disease through the development and delivery of medications that address the urgent unmet need and fill a therapeutic void. Through continued clinical evaluation of investigational therapies, we’re fulfilling our promise to these communities.

Left untreated, these diseases can lead to a host of life-altering symptoms that can profoundly impact the quality of life for patients and their families.

The patient communities deserve better. We believe our approach can address some of the most burdensome symptoms associated with these rare and life-threatening diseases.

Learn more about our treatments for Alagille syndrome, progressive familial intrahepatic cholestasis and for certain kinds of Bile Acid Synthesis Disorders


We proudly serve the children, adults, and families with rare disease, working closely with these communities to intimately understand the challenges they face day to day over time. It is with this deep understanding, empathy, and urgency that we seek to develop remarkable therapies for these rare diseases that have limited or no approved treatment options. Because ultimately, our goal is to transform the lives of these individuals so that they have the opportunity to live life to the fullest.

LEARN MORE ABOUT OUR THERAPEUTIC AREAS OF INTEREST