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…
Cholestasis
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…
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…
