I was primary caregiver for my mother. For about 5 years.Others casually speak of the conventional red tape of the FDA holding this back for ten years, but there are just too many people and their children and grandchildren who are affected, and too devastatingly, for business as usual to fly.
"Safety" is a red herring issue when the disease is fatal, and catastrophic for the family before the actual fatality. The only serious issue is effectiveness. You'd trade five years of living death for one year of clarity in a heartbeat.
Good post, great summation.
Something has to be done to protect the manufacturer in the event something goes wrong though.
This would be nothing more than an enormous experiment.