I found this article in the New Yorker when I was reading a more recent one. One of a Kind - What to do if your child has a condition that is new to science? Seth Mnookin The takeaways for getting progress on a rare disease seem to be: it helps to have plenty of money, to access researchers and fund their further work social media can have a big impact on progress It was interesting to read the following, bearing in mind that this was in 2014, when only around 9 children with this particular genetic condition had been identified across the world.
