Livmarli benefits seen across PFIC types, trial data show
Treatment with Livmarli (maralixibat) leads to significant clinical benefits for people with progressive familial intrahepatic cholestasis (PFIC) across a wide range of disease…
Cholestasis
New mutations tied to rapidly progressing intrahepatic cholestasis
Researchers in China have identified five new mutations in the NR1H4 gene in three boys with progressive familial intrahepatic cholestasis type 5 (PFIC5) and…
Low-dose soybean emulsion may avoid newborn cholestasis: Study
A reduced dose of soybean fat emulsion seems to be better than the standard dose at preventing parenteral nutrition-associated cholestasis in newborns undergoing surgery…
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…
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…
