This is why gene therapy can be dangerous.
However, given how horrible the disease is, I’d still try to cure it even if some people died from the therapy itself.
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Ah - they don’t even know why he died. I don’t think we have enough information yet to draw any conclusions from this situation. Lets wait to see how it all comes out after they report more before we draw any conclusions.
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True, but for a toddler to die, I think it has to be related to the disease and/or the failure of the treatment (probably not old age). If you read between the lines, you’ll see they’ve stopped all further treatment of new patients. This is damage control.
On the other hand, the disease is horrible and if you needed to choose between a long lingering death from this debilitating disease vs a chance of success of a cure through genetic therapy vs a quick death from a therapy that doesn’t work, if I was the parent, I’d still try the therapy.
This comment on the article rang true for me:
My nephew has Duchenne. We all know the horrible end of the story. My brother will do anything in his power to have genetic therapy administered. We are trying to raise the 4 million dollars needed. We know death or any side effect is possible. However, a slow and painful death is coming to him nonetheless. So please stop blaming Pfizer, we are adults and understand the dangers we will be facing. If you are not living in this nightmare, please remain silent, and let science try to progress, we need a cure, and will take any risks to have it.
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Duchenne is awful. Hence on a risk/reward basis dangerous experimental work is warranted.
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I read about this this morning. Sad, but I do not believe the treatment is irresponsible in any way.
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Next up should be removing 5ar type 2 production on the scalp and prostate 
Pfizer determined the gene therapy does not work, and all development has been halted.
June 12 (Reuters) - Pfizer said on Wednesday its experimental gene therapy for Duchenne muscular dystrophy (DMD) failed to improve the motion function of patients in a late-stage trial compared with a placebo.
The therapy also did not show a significant difference compared to placebo in secondary goals of the study such the time it takes for patients to rise from the floor or improve speed in a 10-meter run or walk.
I would generally stay away from anything from Pfizer until it has been in the market for 10 years. The amount of lawsuits they have is staggering…
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