Type of intrahepatic cholestasis shows abnormal cellular processes
The levels of more than 250 proteins are abnormal in lab-grown liver organoids, or mini-livers, derived from a mouse model of progressive familial intrahepatic…
Cholestasis
Navigating the unknowns after a diagnosis of PFIC
One difficult thing about a rare disease is that there are so many unknowns, and it’s not always obvious where the answers can be found.
New NR1H4 mutation identified in baby girl with PFIC5
A new mutation in the NR1H4 gene was identified as the cause of progressive familial intrahepatic cholestasis type 5 (PFIC5) in a baby girl…
Nonprofit funds research to test for pregnancy complication ICP
A nonprofit group has donated $60,000 to support researchers at Yale University School of Medicine working to expand access to a new test that can…
Brain changes seen in children with neonatal cholestasis: Study
Brain scans have shown children with neonatal cholestasis tend to have brain abnormalities, a review study found. Bleeding was the most common, especially in…
Livmarli led to ‘dramatic responses’ in PFIC trial, Mirum exec says
The Phase 3 trial that supported the U.S. approval of Livmarli (maralixibat) for progressive familial intrahepatic cholestasis (PFIC) involved the broadest range of patients,…
Worse outcomes risk rises with liver ailment ICP, gestational diabetes
Having gestational diabetes increases the risk of adverse outcomes in pregnancy for women with intrahepatic cholestasis of pregnancy (ICP), the most common pregnancy-related liver…
FDA approves Mirum’s Livmarli for PFIC patients ages 5 and older
The U.S. Food and Drug Administration (FDA) has approved Mirum Pharmaceuticals’ Livmarli (maralixibat) to reduce pruritus, or itching, in adults and children, 5 years…
Sodium 4-phenylbutyrate delayed transplant need in 2 PFIC2 children
Treatment with an oral medication called sodium 4-phenylbutyrate — approved to treat diseases in which the body fails to remove a waste called ammonia…
Probiotics may help women with ICP, but risks not yet known
Women with intrahepatic cholestasis of pregnancy (ICP) show lesser diversity across their gut bacteria than do other pregnant women, a study reports. Its findings…
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