>>7304183 (PB)
>I posted this the other day with no traction.
>They have a cure for Child Cancer.
>The COST to patients is beyond belief.
>https://www.wsj.com/articles/at-2-million-new-novartis-drug-is-priciest-ever-11558731506
>The world’s most expensive medicine is about to hit the market.
>A one-time treatment for a devastating infant muscle-wasting disease won approval from the U.S. Food and Drug Administration Friday. Its maker Novartis AG NVS 0.80% says the gene therapy will cost $2.125 million.
>The therapy, called Zolgensma, treats an inherited disease called spinal muscular atrophy, or SMA, whose victims typically die before the age of two if untreated. It is the latest gene therapy—a technique that introduces new DNA into the body to correct a faulty gene—to win approval.
Yeah, I had to give a lecture on SMA last year
SMA is a rare genetic disease caused by the deletion of the human gene called smn1 gene. “SMN” stands for “spinal motor neuron” and was named that because in humans born without the gene experience progressive death of spinal motor neurons eventually causing death. But here is the thing, back in the 70’s before the gene was known, it was found that these neurons die because of a deficit in L-carnitine, normally synthesized in muscles. When L-carnitine was provided by dietary supplementation, the clinical symptoms in human patients improved but these studies were never followed up on (too easy?). The SMN1 gene is also involved with the “splicosome” <sp?an “epigenetic regulator” which means that there are likely numerous ways one could correct the disorder without needing the gene. For example, in a mice model of SMA, researchers found that an old anti cancer drug, which is a pro-drug of butyric acid (found in butter), when given early during a critical period, life span could be extended by 250% without needing the SMN1 gene.
In the age of epigenetics, the “central dogma” of DNA——>mRNA——>Proteins is now dead, but those biochemically indoctrinated will not let go. They will say spending 2.5 million to treat a disease is a huge triumph for gene therapy. It’s complete bullshit and such monopolized therapies are entirely unnecessary.