Mirum Pharmaceuticals Inc.

The sponsor also funds research in rare pediatric cholestatic liver diseases, especially Alagille syndrome and progressive familial intrahepatic cholestasis. The clinical program addresses long-term disease management, symptom burden, and liver-related outcomes in affected children and adolescents.

• Pediatric cholestatic liver disease
• Long-term safety
• Clinical outcomes
• Growth and development

Research in this area includes monitoring of liver function, cholestasis biomarkers, and complications associated with progressive disease.

Mirum’s trial portfolio includes extended clinical follow-up in chronic liver disease settings, with attention to tolerability, durability of response, and ongoing symptom control in patients treated for cholestatic conditions.

• Liver toxicity monitoring
• Portal hypertension
• Liver fibrosis
• Liver transplant status

These studies reflect interest in disease progression markers, hepatic complications, and long-term therapeutic management across rare liver disorders.

In addition to hepatology-focused programs, the sponsor supports research in Fragile X syndrome, a neurodevelopmental disorder. This work evaluates treatment tolerability and safety in male participants affected by the condition.

• Neurodevelopmental disorders
• Safety and tolerability
• Treatment-related adverse events

This area expands the sponsor’s clinical interest beyond liver disease into targeted investigation for inherited neurological conditions.