familial chylomicronemia syndrome

fa·mil·i·al chy·lo·mi·cro·ne·mi·a syn·drome

an inherited disorder resulting in accumulation of chylomicrons as well as triacylglycerols.
See also: chylomicronemia.
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The most advanced drug in its pipeline, volanesorsen, has successfully completed a Phase 3 clinical program for the treatment of familial chylomicronemia syndrome, or FCS, and is currently in Phase 3 clinical development for the treatment of familial partial lipodystrophy, or FPL.
Drugs currently in Phase 3 development include volanesorsen, a drug Ionis is developing and plans to commercialize through its wholly owned subsidiary, Akcea Therapeutics, to treat patients with either familial chylomicronemia syndrome or familial partial lipodystrophy; and IONIS-TTRRx, a drug Ionis is developing with GSK to treat patients with TTR amyloidosis.
The product is intended for the treatment of patients with Familial Chylomicronemia Syndrome.
Presently, the company is conducting an international multi-centre, randomised, double-blind, placebo-controlled Phase three study in patients with Familial Chylomicronemia Syndrome.
i) Familial Chylomicronemia Syndrome with either Lipoprotein Lipase (LPL) Deficiency or Apolipoprotein C II deficiency or
M2 PHARMA-March 7, 2017-Akcea, Ionis Announce Tout Positive Results from Study of Volanesorsen in Familial Chylomicronemia Syndrome
The pivotal Phase 3 APPROACH study of volanesorsen met its primary endpoint of reducing triglyceride levels in patients with familial chylomicronemia syndrome, US-based RNA-targeted drug discovery and development company Ionis Pharmaceuticals, Inc.
focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders, today announced that the company will present new data on the clinical characteristics, impact on quality of life and psychosocial consequences of living with familial chylomicronemia syndrome (FCS).
M2 EQUITYBITES-July 7, 2015-Akcea Therapeutics wins US FDA's orphan drug designation for Volanesorsen for treating Familial Chylomicronemia Syndrome
Drug company Isis Pharmaceuticals (NasdaqGS:ISIS) announced on Thursday that it has initiated a Phase 3 study evaluating ISIS-APOCIIIRx in patients with familial chylomicronemia syndrome (FCS), a rare orphan disease that is characterised by extremely high triglyceride levels.
M2 PHARMA-July 7, 2015-Akcea Therapeutics wins US FDA's orphan drug designation for Volanesorsen for treating Familial Chylomicronemia Syndrome

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