Video
Author(s):
This past year, James A. Underberg, MD, MS, FACPM, FACP, FASPC, FNLA, President of the National Lipid Association (NLA), outlines familial chylomicronemia syndrome (FCS) and the need for treatments, which may not be far.
This past year, James A. Underberg, MD, MS, FACPM, FACP, FASPC, FNLA, served as President of the National Lipid Association (NLA). While at the NLA’s 2018 Scientific Sessions in Las Vegas, Nevada, he took time to outline familial chylomicronemia syndrome (FCS) and the need for treatments, which may not be far.
Underberg: For those of us that take care of patients with FCS, there really isn’t much we can do for our patients right now. For patients who are afraid to ever eat outside their own home, can’t go out to someone’s home for dinner, can’t travel, and worry every time they put a piece of food in their food they might end up in the emergency—I think a drug like this might be life-saving. Certainly, for those of us who care for these patients, it’s difficult because we don’t have anything right now to offer them.
While they represent a relatively small group of patients with a rare disorder, about one in a million, they still find their way to practitioners, like myself, who are clinical lipidologists. Having something to offer them, I think, will be very much like what happened five years ago when patients of homozygous familial hypercholesterolemia had only one option, which was apheresis. Now there are multiple treatment options for patients with homozygous FH to avoid apheresis, and I’m hoping in the future that might be the case for FCS.
For more, follow Rare Disease Report on Facebook and Twitter.