Saturday, April 20, 2013

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



On Tuesday January 29th, the Food and Drug Administration approved a new drug, Kynamro, which treats high cholesterol and heart attacks. Kynamro can also shut off specific genes that cause the disease.
Kynamro, known generically as mipomersen, inhibits action of a gene, apolipoprotein B; that is involved in the formation of particles that carry cholesterol in the blood.

Kynamro was invented by Isis Pharmaceuticals and will be marketed by Sanofi’s Genzyme division. Isis has been pursuing antisense technology since the company’s founding in 1989. Antisense drugs work essentially by shooting the messenger. The recipe to make a protein is carried from a gene in the nucleus into the body of a cell by a single strand of RNA, called messenger RNA. Antisense drugs are tiny fragments of synthetic DNA or RNA that bind to mRNA that could inactivate or destroy it.

Kynamro has some worrisome side effects such as homozygous familial hypercholesterolemia or HoFH, liver damage, and flulike symptoms.

 

1 comment:

  1. It is great that they found a new medicine to treat high cholesterol and heart attacks, but at what cost is it to take this medicine? When the side affects are just as bad as the things they are treating it puts the person in a bind of whether to cure what they have wrong and potentially get something just as bad or go on living with high cholesterol and high risk of heart attack. Besides that it is still a good new find in the way it can shut off specific genes that causes cholesterol.

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