First successful treatment for progeria!!!

I was so happy to hear this news this morning when my alarm woke me up! http://www.medicalnewstoday.com/articles/250725.php

This is really huge, Mayra! Once I read the read the article, I saw how huge this really is:
These kids have a genetic mutation that produces the protein progerin, which is responsible for the disease. Progerin blocks normal cell function and causes harm to the body via a farnesyl group, a molecule that attaches itself to the protein. FTIs prevent this attachment from happening.
http://www.medicalnewstoday.com/articles/250725.php

75% of all kids with progeria in the world took part in this drug took part in this drug trial. I remember when it started. I saw a show about a little English girl and some other kids waiting to see if they were accepted into the program. I know some of them were and they were asking donations to fly to the U.S. , IIRC.

It seems that with this drug, if they can give it to newborns showing signs of the disease, they could stop it.

Yeah, it was a documentary about a little girl named Hayley Okines, I think. Here may be a clip (although it could be from an earlier documentary about the same kid): [ame="http://www.youtube.com/watch?v=tdCwuvp4-p0"]The Child Who's Older Than Her Mother - YouTube[/ame]
 
What wonderful news!! I have seen many children with this condition on various TV shows and am just thrilled to hear that there is progress being made.
 
i knew a little boy in my church that had this. at the time he was one of the oldest children to have this. i moved and lost track of them but often wonder what happened. so glad this progress is being made!!!
 
I've been fascinated with this disorder since I first heard of it many, many years ago. I remember this story on one boy who played the part of an alien, I believe, in a famous movie (for the life of me, can't remember which), and how after the day's shoot, the actors (one a grizzly, mountain man type) would sit with the boy and play poker, smoke cigars, and have the occasional scotch. This kid was obviously a minor, but the mom's take was that he had such a short time left in the world, why not let him experience everything he could while he was physically able. I doubt he's still alive, but I can't help hoping he is still around to maybe benefit from this new discovery.
 

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