Genetic Diseases News

The FDA has approved Ctexli for the treatment of cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease, in adults.

In a Healio video exclusive, Pam Vig, PhD, highlights new data on Livmarli for the treatment of patients with pruritus due to progressive familial intrahepatic cholestasis and Alagille syndrome, which were presented at EASL Congress.

Healio will present its sixth annual Disruptive Innovators Awards for gastroenterology and hepatology this fall.

Arrowhead Pharmaceuticals announced that the first patient has been treated in Takeda Pharmaceuticals’ phase 3 clinical trial of fazirsiran for the treatment of alpha-1 antitrypsin deficiency associated liver disease.

LONDON — Long-term givosiran treatment provided sustained benefit and improved quality of life among patients with acute hepatic porphyria, according to research presented at the International Liver Congress.

A kidney disease panel for 382 genes yielded a high success rate and was effective in identifying monogenic variants underlying inherited kidney diseases, according to data published in the American Journal of Nephrology.

In this exclusive video, Andrew Allegretti, MD, MSc, discussed a post hoc analysis assessing serum creatinine in patients treated with terlipressin for hepatorenal syndrome-acute kidney injury.

Transforming growth factor beta mutation biomarkers may help identify patients at high risk for cancer of the liver, pancreas and gastrointestinal system, according to data published in Gastroenterology.

Alnylam Pharmaceuticals Inc. reported new data from the open-label extension period of the Envision phase 3 study that further demonstrated the long-term therapeutic benefit of Givlaari in patients with acute hepatic porphyria.