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(chylomicronemia syndrome)
145 results
  • Spectrum of Mutations and Long-Term Clinical Outcomes in Genetic Chylomicronemia Syndromes. [Journal Article]
    Arterioscler Thromb Vasc Biol 2019; :ATVBAHA119313401D'Erasmo L, Di Costanzo A, … Arca M
  • CONCLUSIONS: Our data indicate that the genetic architecture and natural history of FCS and MCS are different. FCS expressed the most severe clinical phenotype as determined by resistance to triglyceride-lowering medications and higher incidence of acute pancreatitis episodes. The most common genetic abnormality underlying FCS was represented by biallelic mutation in LPL while APOA5 variants, in combination with high rare polygenic burden, were the most frequent genotype of MCS.
  • Successful Strategies for Mitigation of a Preclinical Signal for Phototoxicity in a DGAT1 Inhibitor. [Journal Article]
    ACS Med Chem Lett 2019; 10(8):1128-1133Harrison TJ, Bauer D, … Patel SJ
  • Diacylglycerol O-acyltransferase 1 (DGAT1) inhibitor Pradigastat (1) was shown to be effective at decreasing postprandial triglyceride levels in a patient population with familial chylomicronemia syndrome (FCS). Although pradigastat does not cause photosensitization in humans at the high clinical dose of 40 mg, a positive signal was observed in preclinical models of phototoxicity. Herein, we desc…
  • Volanesorsen: First Global Approval. [Journal Article]
    Drugs 2019; 79(12):1349-1354Paik J, Duggan S
  • Volanesorsen (Waylivra®), an antisense oligonucleotide inhibitor of apolipoprotein CIII (apoCIII) mRNA, is being developed by Ionis Pharmaceuticals through its subsidiary company, Akcea Therapeutics, to treat familial chylomicronemia syndrome (FCS), hypertriglyceridemia and familial partial lipodystrophy (FPL). In May 2019, volanesorsen was approved in the EU for the treatment of adult patients w…
  • Volanesorsen for treatment of patients with familial chylomicronemia syndrome. [Review]
    Drugs Today (Barc) 2018; 54(12):721-735Warden BA, Duell PB
  • Familial chylomicronemia syndrome (FCS) is a rare autosomal recessive disorder typically caused by mutations in genes for lipoprotein lipase (LPL), apolipoprotein C-II (Apo-CII), apolipoprotein A-V (Apo-AV), lipase maturation factor 1 (LMF1) and glycosylphosphatidylinositol-anchored high-density lipoprotein-binding protein 1 (GPI-HBP1). FCS is associated with severe morbidity that includes recurr…
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