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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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