Rare Disease Report

NLA Scientific Sessions Preview (FCS, HoFH)

APRIL 29, 2014
RDR Staff

The upcoming National Lipids Association (NLA) Scientific Sessions in Orlando will be a showcase for new scientific and clinical data on a number of rare lipid conditions, including homozygous familial hypercholesterolemia (HoFH) and familial chylomicronemia syndrome (FCS). The conference will also highlight some of the new treatment ideas being discussed by experts in these diseases.  
Rare Disease Report will be in attendance to provide conference coverage.
The conference begins Thursday afternoon with noteworthy presentations by Jonathan Cohen, PhD discussing the history of PCSK9 and Raul Santos MD, PhD explaining the differences between HoFH and heterozygous familial hypercholesterolemia (HeFH).
The first evening will conclude with the symposium “New Avenues in the Management of Hypertriglyceridemia” with presentations by Ira Goldberg, MD, Peter Toth, MD, and James Underberg, MD. Daniel Gaudet, MD, will chair the symposium.
On Friday, the focus will be on many of the poster presentation dealing with rare disease. These include
  • 107: Diacylglycerol acyltransferase 1 (DGAT1) inhibition as a metabolic regulator: clinical benefits of pradigastat in obese patients with type 2 diabetes
  • 119:  Phase 1 Single and Multiple Ascending Dose Study of CAT-2003, a Novel Activator of Lipoprotein Lipase, Decreases Postprandial Triglycerides
  • 121: Association between fasting triglycerides and presence of fasting chylomicrons in patients with severe hypertriglyceridemia
  • 123: Diagnosis and treatment patterns of real-world patients with severe hypertriglyceridemia
  • 124: Lipid management and physician visit patterns in a real-world sample of patients with severe hypertriglyceridemia
  • 125: Incidence and characteristics of acute pancreatitis in a real-world sample of patients with severe hypertriglyceridemia
 While Friday’s posters will focus in FCS, Saturday and Sunday we are looking forward to two oral presentations dealing the HoFH: 
  • The Role of Microsomal Triglyceride Transfer Protein (MTP) Inhibition on the Management of HoFH by Pamela Morris MD, FNLA
  • Management Crossroads in the Patient with Homozygous FH by Carl Orringer MD, FNLA
Rare Disease Report will be in attendance at the meeting and will reporting back with the highlights in the coming week.

Image of conference logo from the National Lipid Association.

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