In this text I propose to consider in detail 2 known drugs from SMA.
Zolgensma is the first drug for gene therapy of spinal muscular atrophy. Manufactured by AveXis (Novartis). Therapy provides a functional copy of the SMN gene to stop disease progression through sustained expression of the SMN protein. A functional copy of the SMN gene is administered using adeno-associated virus (AAV) serotype 9, AAV9, which is able to cross the blood-brain barrier and penetrate into the patient's cells without altering the existing DNA. The drug is administered once as an intravenous infusion lasting 60 minutes. Systemic corticosteroids are given to all patients before and after Zolgensma infusion. The most common side effects after treatment are elevated liver enzymes and vomiting. Approved for the treatment of pediatric patients under 2 years of age with spinal muscular atrophy type I and II: in the US, EU, Japan and Brazil. Price $ 2,300000.
Spinraza is the first drug to treat spinal muscular atrophy. The drug was developed by Biogen. Trade name Spinraz. Approved for use: USA, Europe, Russia. Price $ 1,500,000. The drug is administered continuously.
Of course, drugs for such genetic diseases are good. If not one "but". Where, the average family with a special child, get $ 2,300000 and $ 1,500,000? This requires: collecting charitable contributions, selling a car with a home and your own organs. The impression is that the scientists who invented the vaccine specifically set such a price on the market that they bought only the rich or did not buy at all, because the drugs do not work. I believe that for such a price a child should not only be healthy, but also become a minimum of Spider-man and a maximum of Superman. Because drugs work at the genetic level.
