An experimental antisense oligonucleotide (ASO) therapy designed to boost production of the Jagged-1 protein — which is known as Jag1…
Articles by Lila Levinson, PhD
Children in Mexico living with the complex genetic disease Alagille syndrome have limited access to essential care, including confirmatory…
Treatment with Livmarli (maralixibat) quickly and safely reduced severe itching in a 16-year-old boy with Alagille syndrome after his…
High blood pressure is a leading cardiometabolic risk factor — a condition linked to problems such as heart disease, stroke,…
Researchers identified seven genes that may contribute to biliary atresia, a discovery that could provide opportunities to develop treatments…
As part of a merger deal, Roche will acquire 89bio and its treatment candidate pegozafermin — an injection therapy now…
Nesfatin-1 (NF-1), a hormone-like molecule that may ease cellular stress, reduced injury to the placenta in a mouse model of…
The U.S. Food and Drug Administration (FDA) has granted orphan drug and rare pediatric disease designations to ART4, an experimental…
While different types of progressive familial intrahepatic cholestasis (PFIC) vary in their genetic cause and clinical profile, new medications…
Problems in mitochondria, the cell’s powerhouses, may contribute to liver damage associated with cholestasis and represent a potential therapeutic…