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by justrealist
880 days ago
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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). |
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