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Saving Eliza: One family's battle to save young daughter

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  • jenkers Salt Lake City, UT
    April 8, 2014 10:17 p.m.

    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.

  • Kralon HUNTINGTON BEACH, CA
    April 8, 2014 6:05 p.m.

    "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).

  • Irony Guy Bountiful, Utah
    April 8, 2014 5:34 p.m.

    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?