Genetic Diseases News

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.

Arrowhead Pharmaceuticals announced it has discontinued development of three drugs currently being investigated in clinical trials for the treatment of various forms of liver disease administered via the company’s EX-1 delivery vehicle: ARC-520, ARC-521 and ARC-AAT. All patient recruitment for clinical trials have been halted and dosing discontinued.

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 disease, a progressive, irreversible, chronically debilitating and fatal genetic disease, according to the drug’s manufacturer.

The FDA granted PhaseRx Inc. orphan drug designation for PRX-OTC for the treatment of the rare liver disease ornithine transcarbamylase deficiency in children, according to the company.

The FDA granted Biotie Therapies Corporation orphan drug designation for BTT1023 to treat primary sclerosing cholangitis, according to a press release.

In the LOCKCYST I clinical trial, a low dose of Somatuline Depot injection for the treatment of polycystic liver disease was found to be safe and effective, according to published findings. 

In an analysis of placebo-controlled clinical trials, somatostatin analogues improved health-related quality of life among patients with polycystic liver disease, according to study data.

ARC-AAT has been granted orphan drug designation by the FDA for the treatment of liver disease associated with alpha-1 antitrypsin deficiency, according to Arrowhead Research Corporation, manufacturer of the drug.

MRI may be accurate in ruling out hepatic iron overload but not accurate enough to provide a definite diagnosis, according to recent study data.

Members of the All-Party Parliamentary Hepatology Group have asked the National Health Service in the United Kingdom to show more concern about the rise of liver disease.