Individuals with a mutation in this gene tend to have lower amounts of fatty triglycerides and cholesterol in their blood – without exhibiting other kinds of health complications – and for years scientists have been attempting to recreate the method, with treatments that effectively mimic the consequences of the mutation. Still, Evkeeza is a “first of its kind therapy and works on a completely totally different pathway” from PCSK9 drugs, Regeneron’s spokesman said. Along with needing a number of medication, many HoFH patients and their families routinely journey for lipid apheresis treatments to cleanse the cholesterol from their blood, Wilemon mentioned. Underdiagnosis can also be a difficulty, and Wilemon said a new drug launch might help increase awareness concerning the disease. Right now, though quite a few therapies exist to decrease LDL cholesterol, many HoFH patients cannot get their levels to a healthy vary, FH Foundation founder and CEO Katherine Wilemon stated in an interview. Regeneron’s Evkeeza scored approval on Thursday as an add-on to other lipid-lowering therapies in patients 12 and older with HoFH, which impacts approximately 1,300 patients in the U.S.
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