Genetic Diseases

Top Story

Kanuma improves liver markers in patients with lysosomal acid lipase deficiency

Kanuma improves liver markers in patients with lysosomal acid lipase deficiency
December 6, 2016

BOSTON — Kanuma improved various liver markers, as well as reduced liver fibrosis and cirrhosis, in children and adults with lysosomal acid lipase deficiency, according to long-term results of the phase 3 ARISE clinical trial presented at The Liver Meeting.

“The drug is very effective in both reducing inflammation in the liver and improving dyslipidemia which we expect to be translated to reduced cardiovascular risk. These improvements persisted over a period of 76 weeks so there are lasting effects,” Barbara K. Burton, MD, professor of pediatrics, Northwestern University Feinberg School of Medicine, and attending physician at the Ann and Robert H. Lurie Children’s Hospital of Chicago, told

Arrowhead discontinues multiple drug candidates for liver disease

December 1, 2016
Arrowhead Pharmaceuticals announced it has discontinued development of three drugs currently being investigated in clinical trials for the treatment of various forms of…
FDA News

FDA grants rare pediatric disease label to investigational drug for NPC

November 30, 2016
The FDA has issued rare pediatric disease designation to an investigational gene therapy product candidate for the treatment of children with Niemann-Pick Type C1…
FDA News

FDA grants orphan drug designation to PRX-OTC for rare liver disease

November 28, 2016
The FDA granted PhaseRx Inc. orphan drug designation for PRX-OTC for the treatment of the rare liver disease ornithine transcarbamylase deficiency in children
More Headlines »

CRITICAL ISSUES IN HBV: Considerations in Optimizing HBV Treatment

This activity is supported by an educational grant from Gilead Sciences Medical Affairs.

Hepatitis B virus (HBV) infection is a global epidemic with a worldwide burden. Physicians must address the importance…
More »
GI Bookshelf

Chapter 9: Gastroduodenal Stents

From Self-Expanding Stents in Gastrointestinal Endoscopy
More »
Resource Centers
International Liver Congress

International Liver Congress

Meeting News

VIDEO: Expert reviews steps for women physicians to advance careers

May 10, 2017
More »