Livmarli now approved in EU to treat adults, children with PFIC
Livmarli (maralixibat), an oral medication from Mirum Pharmaceuticals, is now approved in the European Union to treat progressive familial intrahepatic cholestasis (PFIC) in…
Cholestasis
Baby with rare PFIC type thrives after liver transplant: Case study
A living donor liver transplant successfully managed liver failure in a 6-month-old boy with progressive familial intrahepatic cholestasis type 8 (PFIC8) that was unresponsive to…
Maternal jaundice in ICP tied to worse pregnancy outcomes: Study
Women who develop a subtype of intrahepatic cholestasis of pregnancy (ICP) marked by jaundice — a yellowing of the skin or whites of the…
Our journey to having a healthy child with IVF
Alongside our daughter Eva’s diagnosis of progressive familial intrahepatic cholestasis (PFIC), we learned that our future children would have a 25% chance of having…
Abuse suspected for skin bruising in case of PFIC2 liver disease
An 8-month-old girl with type 2 progressive familial intrahepatic cholestasis (PFIC2) who was seen at a hospital emergency department with extensive skin bruising was suspected…
Intrahepatic cholestasis of pregnancy risks mothers, newborns
Pregnant women with intrahepatic cholestasis of pregnancy (ICP) have an increased risk of other pregnancy-related problems, including gestational diabetes and preeclampsia, or sudden high…
EMA committee favors approval of Livmarli for PFIC in EU
Note: This story was updated July 9, 2024, to clarify Livmarli is approved in the U.S. for all PFIC forms, but not recommended for a…
Benefits of Livmarli for cholestasis sustained up to 2 years: Trial data
Up to two years of daily treatment with Livmarli (maralixibat) controlled itch and reduced blood levels of bile acids and bilirubin, two markers of…
Blood scores may help with ICP diagnosis, management: Study
Two scores calculated from blood biomarkers can accurately discriminate between pregnant women with intrahepatic cholestasis of pregnancy (ICP) and those without, and predict ICP-associated…
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…
Recent Posts
- How I’m learning to cope with challenges as a rare disease parent
- IBAT inhibitors have potential to boost life quality for patients
- Trial of HBV treatment candidate now enrolling final patient group
- Cour’s CNP-104 granted FDA orphan drug status for treating PBC
- Maralixibat Phase 3 trial for cholestasis-related itch is enrolling