[DoreenMichele]

So far the evidence doesn’t show anything near this level of harm. I’m just skeptical of anything that seems too good to be true.

People with rare diseases (and their families and caretakers) get excited about anything at all actually helpful, especially if it comes through official, approved medical channels, because they suffer a whole helluva lot every single day while listening to the medical community promise "cures" that never seem to arrive. It's an amazingly hard psychological thing to live with.

On a Cystic Fibrosis list years ago, a parent said "My child turns 18 today. The life expectancy now is 36. When he was born, the life expectancy was 18."

So gains get made but these are people who need a miracle cure and needed it "yesterday" who are, instead, getting incremental improvements at what feels like a glacial speed while they live tormented lives.

So, yeah, they get excited. But that doesn't mean they are going to all throw caution to the wind and jump on the band wagon. Some that do -- or seem to -- are basically "already dead anyway" if they don't try something new and are happy to take a gamble in hopes of one last hurrah and at least it's more data for others like them if it doesn't help them.