"Small n" is not always a problem. They are not trying to tease out a small effect size between two treatments. This is a strong signal -- literally children can now hear when they couldn't before. A small sample size does not allow statistical noise to produce that effect.
Yeah the FDA is also being pretty lenient about allowing single-arm trials for gene therapy for this reason. The effect size needs to be obvious, but there's no reason to subject 3/6 deaf children to being a control if the risk is relatively low and the odds of success are high.
(also, for many of theses diseases, there's no plan B -- if it's not fixed in early childhood, you can never interpret sound).
(They took off from Europe with 22 kids and did an "infectious relay" in pairs. By the time they got to South American mainland, precisely 21 kids already resolved their cowpox. One last kid was still barely infectious, thus becoming the sole source of the vaccination material.)
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[ 3.8 ms ] story [ 16.2 ms ] threadIt's experimental gene therapy, with very small n, and only looks applicable for certain types of inherited deafness.
(also, for many of theses diseases, there's no plan B -- if it's not fixed in early childhood, you can never interpret sound).
https://en.wikipedia.org/wiki/Balmis_Expedition
(They took off from Europe with 22 kids and did an "infectious relay" in pairs. By the time they got to South American mainland, precisely 21 kids already resolved their cowpox. One last kid was still barely infectious, thus becoming the sole source of the vaccination material.)
Do they start getting irritated by everyday sounds? I’m really curious ..
Gene therapy allows an 11-year-old boy to hear - https://news.ycombinator.com/item?id=39106464 - Jan 2024 (238 comments)