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APOC3 Interference for Familial Chylomicronaemia Syndrome.


ABSTRACT: Patients with familial chylomicronaemia syndrome (FCS) have severe hypertriglyceridaemia due to genetically absent lipolytic capacity. They have a poor response to conventional therapies. To reduce the risk of potentially fatal pancreatitis, the management of FCS relies principally on a strict low-fat diet, which is difficult to follow and compromises quality of life. Targeted reduction of apolipoprotein C-III using new anti-APOC3 agents, such as the short interfering RNA ARO-APOC3, represents a promising approach to correct the severe biochemical disturbance in FCS.

SUBMITTER: Hegele RA 

PROVIDER: S-EPMC9835816 | biostudies-literature | 2022 Nov

REPOSITORIES: biostudies-literature

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<i>APOC3</i> Interference for Familial Chylomicronaemia Syndrome.

Hegele Robert A RA  

TouchREVIEWS in endocrinology 20221102 2


Patients with familial chylomicronaemia syndrome (FCS) have severe hypertriglyceridaemia due to genetically absent lipolytic capacity. They have a poor response to conventional therapies. To reduce the risk of potentially fatal pancreatitis, the management of FCS relies principally on a strict low-fat diet, which is difficult to follow and compromises quality of life. Targeted reduction of apolipoprotein C-III using new anti-<i>APOC3</i> agents, such as the short interfering RNA ARO-APOC3, repre  ...[more]

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