Hey thanks for reading it!
Yes, there are many of us (me included) hard at work trying to give the people suffering with these diseases some sort of treatment, to restore at least some semblance of quality of life.
Each of these diseases posts their own unique challenges, and the amount of work is massive. I am hopeful that technologies like CRISPR can play a role in helping deal with these genetic issues, but at least to my eyes that tech is further off than many would like. At least in any meaningful wide spread use sort of capacity.
Onward we press!