Thursday, Isis announced trials that could get mipomersen approved in the rare genetic disease familial hypercholesterolemia (FH).

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The ENHANCE study was conducted in 720 patients with heterozygous familial hypercholesterolemia, a genetic disease that can boost untreated cholesterol levels above 300.

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The vote was close, with 9 for approval and 6 against, and only for treatment in a rare genetic disease called homozygous familial hypercholesterolemia (HoFH).

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Such extreme deposition at a young age is akin to that in humans with the rare, genetic form of the disease called familial Alzheimer's.

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Last night, the Food and Drug Administration announced the approval of Kynamro, a new drug to lower cholesterol in patients with homozygous familial hypercholesterolemia (HoFH), a rare genetic disease that causes sky-high cholesterol levels and heart attacks at a young age.

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