Mirum Pharmaceuticals Presents LIVMARLI Clinical Data and Real-World Evidence at NASPGHAN - Seite 2

The full presentations can be found on Mirum’s website within the Publications & Presentations section, and each is also linked within the titles below.

Alagille Syndrome (ALGS)

• Abstract 69: Maralixibat for the treatment of severe xanthomas in two children with Alagille syndrome (poster)
• Abstract 71: Maralixibat persistency and adherence for the treatment of cholestatic pruritus in Alagille Syndrome: Real-world experience in the United States (poster)
• Abstract 72: Maralixibat impact on concomitant medication use for the treatment of cholestatic pruritus in Alagille syndrome: Real-world experience in the United States (poster)
• Abstract 88: Impact of maralixibat on cholestatic pruritus in young adults aged 16 years and older with Alagille syndrome (poster)
• Abstract 89: Maralixibat, an ileal bile acid transporter inhibitor, delays the need for liver transplant in patients with Alagille syndrome: Real-world experience (poster)
• Abstract 626: Real-world safety experience in patients with Alagille syndrome treated with maralixibat (poster)
• Abstract 632: Maralixibat improves xanthomas and hypercholesterolemia in children with Alagille syndrome: An integrated analysis from two clinical trials (poster)

Progressive Familial Intrahepatic Cholestasis (PFIC)

• Abstract 531: Efficacy and safety of maralixibat in patients with progressive familial intrahepatic cholestasis (MARCH): A randomized placebo-controlled phase 3 study (oral presentation)
  **Gerard Odell Prize for Excellence in Pediatric Hepatology Research Recipient**
• Abstract 65: Maralixibat improves cholestatic pruritus and bile acids in children with FIC1: Data from the MARCH trial (poster)
• Abstract 85: Long-term maintenance of response and improved liver health with maralixibat in patients with progressive familial intrahepatic cholestasis (PFIC): Data from the MARCH-ON study (poster)
• Abstract 86: Maralixibat leads to significant reductions in bilirubin for patients with progressive familial intrahepatic cholestasis: Data from the MARCH trial (poster)
• Abstract 347: Analysis of long-term safety in maralixibat-treated participants with progressive familial intrahepatic cholestasis: Data from MARCH-ON (poster)
• Abstract 625: Analysis of safety in maralixibat-treated participants with progressive familial intrahepatic cholestasis: Data from the MARCH trial (poster)
• Abstract 630: Maralixibat leads to significant reductions in pruritus and improvements in sleep for children with progressive familial intrahepatic cholestasis: Data from the MARCH trial (poster)

ALGS and PFIC