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…
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…
Metabolic dysfunction-associated steatotic liver disease (MASLD), a form of fatty liver disease, and other aging-related metabolic disorders may be detectable using computer-based analyses of…
The U.S. Food and Drug Administration (FDA) has approved Vir Biotechnology’s request to conduct clinical testing in the U.S. of a combination of two investigational…
An initiative in the European Union (EU) aims to speed up the diagnosis of rare genetic disorders such as Alagille syndrome in newborns using…
Genetic variations that predispose people toward developing primary sclerosing cholangitis (PSC) are significantly associated with a higher risk of ulcerative colitis, a primary type…
Livmarli (maralixibat), an oral medication from Mirum Pharmaceuticals, is now approved in the European Union to treat progressive familial intrahepatic cholestasis (PFIC) in…
A type of cell death called ferroptosis contributes to metabolic dysfunction-associated steatotic liver disease (MASLD), a form of fatty liver disease that occurs more…
Children with biliary atresia show developmental problems, including language delays, following a living donor liver transplant (LDLT) in infancy and a second surgery to…
High levels of CCL24, the protein targeted by Chemomab Therapeutics’ investigational antibody-based therapy CM-101, are associated with biological pathways contributing to the progression of…
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…