Abstract | INTRODUCTION: AREAS COVERED: We discuss APOC3-targeted experimental treatments for dyslipidemia. There is an emphasis on volanesorsen because it the agent in the most advanced stage of development. M580, a retinoic acid receptor-α specific agonist, an agent in early-stage development is briefly covered. Preclinical data suggest that this agent decreases APOC3 mRNA levels and reduces total cholesterol, TG levels and hepatic lipid accumulation. EXPERT OPINION: The effects of this novel therapeutic approach on cardiovascular morbidity and mortality should be determined in randomized controlled trials. The cost of volanesorsen, the unfavorable safety profile and the need for subcutaneous administration present barriers to long-term use. AM580 may hold promise in the management of hypertriglyceridemia but further investigations are necessary to evaluate safety and efficacy.
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Authors | Dimitrios Milonas, Konstantinos Tziomalos |
Journal | Expert opinion on investigational drugs
(Expert Opin Investig Drugs)
Vol. 28
Issue 4
Pg. 389-394
(04 2019)
ISSN: 1744-7658 [Electronic] England |
PMID | 30753100
(Publication Type: Journal Article, Review)
|
Chemical References |
- Apolipoprotein C-III
- Cholesterol, HDL
- ISIS 304801
- Lipids
- Oligonucleotides
- RNA, Messenger
- Triglycerides
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Topics |
- Animals
- Apolipoprotein C-III
(genetics, metabolism)
- Cholesterol, HDL
(blood)
- Dyslipidemias
(drug therapy, physiopathology)
- Humans
- Hyperlipidemias
(drug therapy, physiopathology)
- Lipids
(blood)
- Oligonucleotides
(adverse effects, pharmacology, therapeutic use)
- RNA, Messenger
(metabolism)
- Randomized Controlled Trials as Topic
- Triglycerides
(blood)
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