Glybera Cures Untreatable Genetic Disorders
Has anybody of you ever thought of the fact, that even the simplest mutation or mis-coding in a gene may cause a serious problem? The innovative drug, Glybera, has been developed to treat a genetic disorder called lipoprotein lipase deficiency (LPLD). More than 1,000,000 people around the world suffer from this genetic disease, caused by a mutated gene that prevents people from making the enzymes necessary to properly metabolize fatty acids in their blood, leading to severe abdominal pain, diarrhea, and acute (and sometimes fatal) pancreatitis. Glybera goes in and modifies your DNA directly, using a deactivated virus to deliver new genetic code into cells, teaching them to properly produce the fat-metabolizing enzymes that they’ve been missing. This drug has been recently approved for use outside of a research hospital or clinical trial, so people will get a possibility to take it in 2013. Of course, Glybera will be very expensive: a series of one-time injections into a patient’s leg muscles will cost insurance companies in Europe $1.6 million per dose. Don’t forget, it’s a new generation of drugs that can treat diseases that were untreatable until now.