Just think how nice it would be if a little girl's life didn't depend
on bake sales or bankrupting her family. Ain't America great?
"But there's a cure, and her family's trying to raise the money to
fund a clinical trial"Actually a clinical trial doesn't
mean there actually is a cure, just a decent hope of a cure. I'm just
mentioning this to be realistic, it is premature to call it a cure.A
clinical trial of a drug has about a 32% of making it to approval. This is
assuming a Phase 1 clinical trial and a Large Molecule drug which is best case
(worst case is small molecule at 13% chance of approval).
Kralon: I believe it's correct as written. You're ignoring all the
intricacies of off-label use, things that are proven in adult populations, etc.,
but not approved for kids, etc. If you follow the links, there's a
discussion of that. The clinical trial would treat her while establishing
efficacy in kids.