Yeah but I don’t like my sister.

quote:

Originally posted by piqaboo:
Its selfish but were it my sister, I'd suggesting waiting for the phase 2 trial.

Actually it’s coming soon so I probably will.

https://clinicaltrials.gov/ct2...ciency&draw=2&rank=1

She says that the man who spoke at the bell-ringing ceremony said that he sat up all night with the box of meds, because it arrived in the afternoon but it has to be taken in the morning. He said that he could literally tell a difference the next morning. He was accustomed to waking up in the night to clear his lungs, but that need went away within days. He called his friends and said, "Is this what you feel like all the time?"

The CEO, who is in his sixties, got all teary-eyed and said that he had to live another twenty years so that he could see what this did for kids who got it from the start.

I agree with you, Jon, for orphan drugs where the numbers are too low to support research. I don't think cystic fibrosis or sickle cell anemia numbers are that low, but I don't know that.

My sister pointed out that the sickle cell treatment will really be revolutionary in Africa, where I presume the numbers aren't low at all.

quote:

Originally posted by jon-nyc:
Did your brother die from it?

In a manner of speaking, he did, though not directly. He suffered from psychosis and was being treated with electroconvulsive therapy. He had a reaction to the general anesthesia and that is what killed him.

quote:

Have you ever considered getting your phenotype checked?

Not until oday, when I read the article I posted. My manual DO has been saying for years she thinks I have a neurological problem but MRIs didn't show anything. When she read in my book that my youngest brother had a degenerative brain disease she seized on that as a clue. But I didn't think I had any symptoms.

I married an Episcopalian, so had no reason to be concerned about being a carrier.

quote:

Originally posted by Mary Anna:
I agree with you, Jon, for orphan drugs where the numbers are too low to support research. I don't think cystic fibrosis or sickle cell anemia numbers are that low, but I don't know that.

In the US the cutoff for orphan drug status is 200k patients, both CF and sickle cell anemia fall well under that. They only count patients in the US.

Oh, wow. That cutoff seems really high.

It’s about 1 in 1500. Europe’s cutoff is 1 in 3000.