Great list! There's also Leber congenital amaurosis, a form of blindness that now has a (very expensive) gene therapy: https://www.wgbh.org/news/2017-10-12/fda

Great list! There's also Leber congenital amaurosis, a form of blindness that now has a (very expensive) gene therapy: https://www.wgbh.org/news/2017-10-12/fda-panel-endorses-gene-therapy-for-a-form-of-childhood-blindness https://www.wgbh.org/news/2017-10-12/fda-panel-endorses-gene...