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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Drugs currently in Phase 3 development include volanesorsen, a drug Isis is developing and plans to commercialize through its wholly owned subsidiary, Akcea Therapeutics, to treat patients with familial chylomicronemia syndrome and familial partial lipodystrophy; ISIS-TTR[sub.
i) Familial Chylomicronemia Syndrome with either Lipoprotein Lipase (LPL) Deficiency or Apolipoprotein C II deficiency or
In addition, we and Akcea plan to complete enrollment in the Phase 3 study of volanesorsen in patients with familial chylomicronemia syndrome this year.
Volanesorsen was granted orphan drug designation from the US FDA for the treatment of patients with familial chylomicronemia syndrome.
Akcea's most advanced program, volanesorsen, is in Phase 3 development to treat patients with ultra-orphan lipid disorders that are characterized by extremely high triglycerides and ApoC-III, including familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy (FPL).
E[sz] Drugs currently in Phase 3 development include volanesorsen, a drug Isis is developing and plans to commercialize through its wholly owned subsidiary, Akcea Therapeutics, to treat patients with familial chylomicronemia syndrome and familial partial lipodystrophy; ISIS-TTR[sub.
Akcea is currently conducting an international, multi-center Phase 3 study of volanesorsen in patients with Familial Chylomicronemia Syndrome (FCS).
This publication follows the December 2014 publication in the NEJM of the positive Phase 2 results from a clinical study of volanesorsen in patients with familial chylomicronemia syndrome (FCS).

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