
Zebrafish used to help save a dying boy - 80mph
https://www.statnews.com/2019/07/01/precision-medicine-zebrafish-helped-save-boys-life/
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vikramkr
There will always be a disconnect between the need for large sample sizes for
rigorous clinical trials and small patient populations for ultra rare
diseases. Hopefully, we can begin developing techniques like this one to
create a robust, validated approach to treat ultra rare diseases without
having to get full phase 1/2/3 FDA approval for each disease case.

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Retric
Their are FDA exceptions for ultra rare diseases.
[https://www.fda.gov/industry/developing-products-rare-
diseas...](https://www.fda.gov/industry/developing-products-rare-diseases-
conditions)

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vikramkr
Even then that's not fully what would be imo an ideal future. Orphan drugs
still require at least a few patients to test on, and these other programs be
more focused on providing loopholes to help out people in tough situations
(very much needed) - but what I'd like to see is rigorous testing and
regulation of a general purpose approach (e.g. FDA approved use of AAV gene
therapy in general for any validated rare disease caused by a single knockout
mutation without having to regulate each vector).

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colordrops
I was expecting something simple like an extract from a zebrafish, but the
treatment in the article is so much more than I expected. Definitely hope this
sort of thing continues to develop.

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cypherpunks01
Paper:
[https://www.nature.com/articles/s41591-019-0479-2](https://www.nature.com/articles/s41591-019-0479-2)

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LeonB
This is a complex story told in simple terms. I think the writer has done a
tremendous job of making the material accessible.

