Hard to keep a head band on.


There are a few interesting tidbits going on in the Rett Syndrome world, mainly some media stuff and awareness raising…

My friend Ingrid, who runs Girl Power 2 Cure, was recently interviewed by Parents TV for a story about her daughter Sarah who has Rett Syndrome. She did an amazing job and I loved seeing all those baby pictures of Sarah and the video of Sarah walking! We saw Sarah in 2008 and she was relying on a stander/walker and not taking independent steps and since then, she’s learned how to walk, pretty fantastic!

There is also a new movie out, which opens tonight, called Extraordinary Measures – which is about a father of two critically ill kiddos with Pompe disease and how he takes a drug from its infancy and gets enough funding to create a biotech business and actually get it through clinical trials and finally being able to find a treatment for his kids. Because critically ill kids often don’t make such great movie material, I was pleasantly surprised when the NY Times gave it a thumbs up.

It’s based on a book called The Cure, which I did spend a few days last week reading through because I was interested in kind of learning how one develops a treatment for a genetic disease. I was a little disheartened to learn via the book that when the kiddos in the movie were diagnosed, there was already in the scientific pipeline at least a few approaches to treating Pompe in animals that were promising. Even with a lot of the science in place, it still took the father (who is a savvy businessman and decidedly not a scientist) of the kids over 5 years to get his kids treated with the medicine. There were issues with funding, scaling up, passing FDA approval, etc, etc. You know, I’m a scientist at heart, so when I think of finding a cure for Rett Syndrome, I think – well OK as long as it’s done in a lab and it works, it’ll be a year before no one will have Rett Syndrome, but perhaps that is not true.

Anyhow, Extrodinary Measures is running a contest: here. The video that gets the most votes gets $10,000. If you could vote for the Rett Syndrome video, that would be great! You can vote everyday until the 29th. The video is a little hard to find, just go to the bottom of the quilt and search for Rett and it’ll pop up.

4 thoughts on “Hard to keep a head band on.”

  1. You gotta go to the "search" option at the bottom of the quilt. Type in Rett and one video will pop up. It's a video with a woman holding a baby in a bed with the hubby smiling broadly.

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