Parents of kids with rare disease found a promising drug

Re:Parents of kids with rare disease found a promising drug by aaronbrethorst on Thursday April 07, 2016 @05:45PM (#11450961)

FTA:

    They found cyclodextrin and initially tried using it in
    oral doses, which is known to be safe. However, the 
    chemical couldn’t effectively reach the brain that way.
    The couple made headlines with their tireless efforts to
    get drug companies, the FDA, and doctors to let them try
    out intravenous treatments of cyclodextrin for their
    twins—and they won. Regular treatments gradually
    improved—although didn’t cure—the twins’ conditions.
    Cyclodextrin is now in clinical trials to treat other
    kids with NPC.

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Re:Parents of kids with rare disease found a promising drug by dekhn on Friday April 08, 2016 @09:47AM (#11454595)

This interesting approach just raises more questions than it answers; experiments with sample size n=1 being run by non-medically trained individuals are unorthodox and generally suspect in the modern medical climate.

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Re:Parents of kids with rare disease found a promising drug by aaronbrethorst on Friday April 08, 2016 @04:29PM (#11457656)

here's another experiment for you that I've run on myself:

When I consume products that contain wheat, I feel sick. When I don't, I feel fine. Sample size n=1, and I am not medically trained. What conclusions, if any, do you think I can draw from my experience?

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Re:Parents of kids with rare disease found a promising drug by dekhn on Saturday April 09, 2016 @10:58AM (#11461508)

I don't know, did you control for placebo effects? Did you verify there isn't something else in those products that makes you feel sick?

Science is hard. Control experiments are key.

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Re:Parents of kids with rare disease found a promising drug by actaeon169 on Friday April 08, 2016 @08:09PM (#11459129)

Clearly, I need to stop eating wheat.

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