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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 Healio.com/Hepatology.

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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…

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Arrowhead Pharmaceuticals announced it has discontinued development of three drugs currently being investigated in clinical trials for the treatment…

FDA News

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The FDA has issued rare pediatric disease designation to an investigational gene therapy product candidate for the treatment of children with…

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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

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