I learned about Tay-Sachs in high school biology. I think we watched a short documentary on it, as an example of genetic inheritance, and the importance of enzyme function. I remember being so surprised that something so simple (absence of one protein) could be so horrible. A beyond-grim prognosis, and immeasurable/unavoidable suffering for everyone involved. Since then, it's been something that I can't reconcile with the existence of a higher benevolent being. I'm no expert, but it made a lasting impression on me.
I'm so uplifted that researchers have made progress on curing this senseless disease.