F.D.A. Approves Genetic Drug to Treat Rare Disease

The New York Times reports that “The Food and Drug Administration approved a new drug Tuesday that not only treats a rare inherited disorder that causes extremely high cholesterol levels and heart attacks by age 30, but does so using a long-sought technology that can shut off specific genes that cause disease.

The drug, Kynamro, which was invented by Isis Pharmaceuticals and will be marketed by Sanofi’s Genzyme division, is unlikely to be a blockbuster. It has some worrisome side effects and there might be no more than a few hundred people in the United States with the disease, known as homozygous familial hypercholesterolemia, or HoFH.”

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http://www.nytimes.com/2013/01/30/business/fda-approves-genetic-drug-to-treat-rare-disease.html?emc=tnt&tntemail0=y

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