Patients & Families

Mirum is focused on developing a safe and effective treatment for children with rare cholestatic liver diseases. What we want, more than anything, is to enable patients and their families to live a healthy and happy life, liberated from around-the-clock disruptions of disease.

We are currently advancing maralixibat for the treatment of progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS), for which there are no approved therapeutics.

Genetic Testing

Mirum Pharmaceuticals is partnering with Retrophin, Inc., to provide, to qualified patients at no charge, the EGL Genetic Cholestasis Panel. This panel may assist in diagnosing a child who has one of these cholestatic liver diseases. For more information about how to access this program, please contact


Hear From Patients, Families & Clinicians

At Mirum, we work closely with patient communities on every part of our business from disease awareness and education to clinical trial design. Hear from patients, families and clinicians for their point of view on the diseases and the need for a transformative medicine.

Clinical Trials

We don’t just work on behalf of patients, we work with them. Through clinical trials for our investigational medicine, maralixibat, patients and their families play an integral role in helping us evaluate the drug and obtain the evidence we need for regulatory approval in the U.S. and beyond.

We initiated the Phase 3 MARCH study of maralixibat in children with PFIC in July 2019. For more information about how to enroll in our clinical trials, please contact us at

Expanded Access

Expanded Access (“Compassionate Use” or “Early Access”) Statement

Clinical development is a critical part of evaluating experimental drugs as potential impactful medicines for children with rare cholestatic liver diseases. Our ultimate goal is the rigorous testing of the experimental drugs with the aim of securing regulatory approval and enabling the medicine to be available to as many patients as possible as quickly as possible.

If you would like more information on our planned Expanded Access Program for Alagille syndrome (ALGS) patients, in the U.S. and Canada, please visit

If you would like more information on our MARCH-PFIC Phase 3 study please visit, or email us at

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