Why weren't they compared to other children who were in similar situations. I'm guessing the population is quite small and variable as it's quite rare but no mention of the standard disease progression.
Would they have died? Where that assumption from? People are making assumptions left right and centre and making investment decisions based on them.
I'm merely pointing out these assumptions aren't valid.
The clinical results may be impressive. Maybe they're not. Again, who knows man.
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