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Isn't that the Lorenzo's Oil disease?

So gente treatment for very serious and fatal disease causes very serious issues in 1/10 of patients?

As opposed to, you know, the effects of the original disease?

I think I would go with this treatment regardless




I should clarify... 7/67 SO FAR

Presumably the other children may manifest cancer down the line. Off target insertion was random

Also the blood cancer has already killed one of the patients


On the other hand, cerebral adrenoleukodystrophy would be expected to kill a significant fraction of those 67 and rob the rest of their cognitive capacity within a few years.

There are times where tolerating treatment-related mortality is the best alternative available.


Which is why these dangerous experimental treatments are used on patients with these classes of terrible diseases first. If it doesn't work or fails horribly it's not much different than what would happen naturally.


> Also the blood cancer has already killed one of the patients

This isn’t a fair interpretation of the death - the treatment of the drug-linked cancer bears a risk of multiple complications. One of these complications led to a patient’s death.


Yeah, like with everything in healthcare. Treatments are almost always harmful in some way, what counts is the benefit/risk ratio.


Side note, did not realize the director of mad max made a movie about this with Nick Nolte and Susan Sarandon in the 90s!




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