Innovative Treatment in Lowering LDL-C Emerges

At last, the Food and Drug Administration has approved a new medication, Kynamro, to help treat individuals suffering with the genetically inherited disease, Homozygous Familial Hypercholesterolemia (HoFH). This condition causes astronomically high levels of one’s Low-Density Lipoprotein Cholesterol. Untreated cases report patients having levels over “1000 mg/dl” in comparison to the healthy standard of 130 mg/dl. Severe heart disease and complications are inescapable for these individuals, and heart attacks usually cause life expectancy to max out in the early 30’s. Cholesterol-reducing Statins and agonizing blood cleansing treatments have always been the standard treatment in the past.



                Thankfully, through the work of scientists at Isis Pharmaceuticals in California, there is now an alternative medication for patients struggling with this disease to try! Kynamro works as an “antisense” drug and literally inhibits a target gene, apolipoprotein B, that is responsible for cholesterol formation.  Creating a functional antisense is an extremely difficult process. It’s designed as

“snippets of synthetic DNA or RNA that bind to that messenger RNA in a way that inactivates or destroys it.”

Clinical trials have shown relatively decent results and an overall decline in HoFH patients’ LDL cholesterol levels. Although injections of Kynamro may cause influenza-like symptoms, the risk may be well worth the reward…

[I wrote this blog using the following article.]

http://www.nytimes.com/2013/01/30/business/fda-approves-genetic-drug-to-treat-rare-disease.html

[For more reference material about the new drug Kynamro, you can also visit the following link.]

http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm337195.htm