What I like about this is its proof of concept nature. Now we know that it is actually possible to prevent at least some parts of an inheritable disease by one-time administration of a drug at the right moment in the development process. That's awesome. There might be other diseases for which that approach might also work. I hope it will spark interest in research into that.
The sad part is ofcourse the one about companies not being interested in drugs for rare diseases, even more when they're not needed frequently. I really hope we can find a solution to that.