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Confidence in gene therapy grows, as GlaxoSmithKline offer their latest treatment Strimvelis, a therapy for severe combined immunodeficiency, with a money-back guarantee
Pharmaceuticals are complicated to produce and test. Metabolic pathways are interactive, fluid constructs that change and adapt to any external influence. This makes targeting any disease with specific pharmaceuticals exceptionally challenging; the body often reacts in undesirable ways, and individual differences can confound studies. Gene therapy is different in many respects – at least when it comes to monogenic disease in which one mutated gene is known to cause a condition. The great thing about gene therapy is not only that it offers permanent cures for these deadly conditions, but that if it works it’s a simpler, more elegant concept of treatment. Fix the gene and you’ve fixed the disease (in many cases anyway). A cure or a refund The first gene therapy drug approved in the West is Glybera, which came in at a whopping $1 million price tag and targets lipoprotein lipase deficiency. GSK’s Strimvelis instead comes in at a considerable 594,000 euros ($665,000), remaining one of the most expensive single therapies in the world but having come down considerably.“The drug has to deliver what you say or we don’t pay,” Luca Pani, director general of the Italian Medicines Agency, told the publication. “If it does not work, they will return the money”
David Vetter, who sadly died at the age of 12 in 1984, was known as the ‘bubble boy’, and was born with severe combined immunodeficiency.
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