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Re:7/67 Children Receiving Skysona Gene Therapy Develop Bloo... by mlyle on Thursday October 10, 2024 @01:33AM (#41795848)
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.

Re:7/67 Children Receiving Skysona Gene Therapy Develop Bloo... by Sabinus on Thursday October 10, 2024 @02:12AM (#41796037)
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.