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An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)

2019-12-10 01:21:16 | BioPortfolio

Summary

The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.

Description

The study will be conducted at multiple sites in North America, Europe, Asia, and South America.

Study Design

Conditions

Progressive Familial Intrahepatic Cholestasis (PFIC)

Intervention

Maralixibat

Status

Not yet recruiting

Source

Mirum Pharmaceuticals, Inc.

Results (where available)

View Results

Links

Published on BioPortfolio: 2019-12-10T01:21:16-0500

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Medical and Biotech [MESH] Definitions

An ATP-binding cassette, sub-family B protein (P-glycoproteins) that functions in the ATP-dependent secretion of BILE SALTS into the BILE CANALICULI of HEPATOCYTES. Mutations in the ABCB11 gene are associated with progressive familial intrahepatic cholestasis 2 (see CHOLESTASIS, INTRAHEPATIC).

Impairment of bile flow due to obstruction in small bile ducts (INTRAHEPATIC CHOLESTASIS) or obstruction in large bile ducts (EXTRAHEPATIC CHOLESTASIS).

Impairment of bile flow due to injury to the HEPATOCYTES; BILE CANALICULI; or the intrahepatic bile ducts (BILE DUCTS, INTRAHEPATIC).

JAUNDICE, the condition with yellowish staining of the skin and mucous membranes, that is due to impaired BILE flow in the BILIARY TRACT, such as INTRAHEPATIC CHOLESTASIS, or EXTRAHEPATIC CHOLESTASIS.

FIBROSIS of the hepatic parenchyma due to obstruction of BILE flow (CHOLESTASIS) in the intrahepatic or extrahepatic bile ducts (BILE DUCTS, INTRAHEPATIC; BILE DUCTS, EXTRAHEPATIC). Primary biliary cirrhosis involves the destruction of small intra-hepatic bile ducts and bile secretion. Secondary biliary cirrhosis is produced by prolonged obstruction of large intrahepatic or extrahepatic bile ducts from a variety of causes.

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