Comments about ‘Saving Eliza: One family's battle to save young daughter’

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Published: Tuesday, April 8 2014 3:30 p.m. MDT

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Irony Guy
Bountiful, Utah

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).

Salt Lake City, UT

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.

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