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.
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).
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.
