Seven new ABCB11 gene mutations identified as cause of PFIC2
Seven new ABCB11 gene mutations were identified as the likely cause of progressive familial intrahepatic cholestasis (PFIC2), according to a study that analyzed the…
Cholestasis
Liver disease in pregnancy linked to worse maternal, newborn outcomes
Intrahepatic cholestasis of pregnancy (ICP), the most common pregnancy-related liver disease, is significantly linked to higher rates of several maternal, fetal, and newborn issues…
Case study supports safety, efficacy of liver transplant for PFIC3
A 3-year-old boy with progressive familial intrahepatic cholestasis type 3 (PFIC3) was safety and effectively treated with a liver transplant from his mother after failing…
PFIC treatment IN016 gets FDA orphan drug status
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to IN016, an experimental therapy Innorna is developing for progressive familial…
Blood biomarkers may aid newborn cholestasis diagnosis: Study
Measuring blood levels of bile acids, a component of bile, and the gamma-glutamyl transferase (GGT) enzyme may aid newborn cholestasis diagnosis by distinguishing between…
Livmarli now approved in US for PFIC patients 1 year and older
The U.S. Food and Drug Administration (FDA) has approved a label expansion for Livmarli (maralixibat) that allows the medication to be used in patients with…
Certain fatty molecules may help diagnose liver disease in pregnancy
Blood levels of certain fatty molecules, called short-chain fatty acids (SCFAs), in pregnant women may help diagnose intrahepatic cholestasis of pregnancy (ICP), according to…
PFIC therapy IN016 earns FDA’s rare pediatric disease designation
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to IN016, Innorna’s experimental therapy that aims to address the root…
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…
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…
Recent Posts
- Abnormal amino acid profile seen in biliary atresia infants after birth
- Our daughter’s PFIC diagnosis made us rethink drinking at Christmas
- Hepatitis D treatment combo granted new designations in US, EU
- Committee recommends seladelpar approval in EU for treating PBC
- Diet rich in omega-3 fatty acids may lower risk of ICP, scientists say