There is an article in this week’s New Yorker talking about new cystic fibrosis drug therapies which enable broken proteins to restore normal function. I hope this is just the beginning of a long a fruitful path which will show that all types of broken proteins can be fixed. Cystic fibrosis is one of the most common rare genetic diseases. Their national organization is well funded and is spearheading a lot of great drug trials and research.
Yes, keep hope alive 🙂