The FDA approved mipomersen sodium (Kynamro), an injection that’s paired with lipid-lowering medications and diet for the treatment of homozygous familial hypercholesterolemia (HoFH). HoFH is an extremely rare condition that often causes heart attacks and even death in young patients. Kynamro is an orphan drug approval that is intended to treat fewer than 200,000 people. Read the FDA's statement.

Keywords: Anticholesteremic Agents, Diet, Hyperlipoproteinemia Type II, Oligonucleotides, Orphan Drug Production, Rare Diseases

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