Hi All,

Well, I am no expert but I on my visit to Dr. New's she did tell me a little  bit about gene therapy.  I'll try to share what I remember.  Basically, she told me that she expects that a cure is about 10 to 15 yrs away.  I lit up when she said this because that would be during my sons' lifetime. She told me that they have a strain of mice that have CAH (or something similar in mice). They are trying to insert the corrected gene into these mice. They are  running into several hurdles. Some of these according to Dr. New and Ann Carlson, who also told me some about the research, is that the mice die very easily.  They are not very hardy little guys. Another hurdle is that they want the correction to last longer than a day or a week. The way to get the new gene into the body is another hurdle.  They can't get funding from the NIH until they show success with the mice trials. So it will be a long hard process but I am thankful that Dr. New is interested in finding a cure for this. Like one mom said to me, "50 years ago, parents were praying for a treatment so their children didn't die, we are so lucky to have a treatment and hope for a cure".

I think that Dr. New talked about gene therapy research at the workshop that Kelly put together. Maybe someone that attending can share.

I keep hoping that someday a cure will be found. I have made it a priority in this new year and all of the years of my life to help raise funds for a cure.  I believe Kelly has a link for donations to research being done by Dr. New.

With that said, about the time released meds. Where to start? I guess those of us who are interested in pursuing this should talk about what exactly we want to ask the drug companies for.  Then I guess we need to start some kind of campaign to get to our accomplishment. Does anyone know of anyone that works for a drug company? They might have some insight into what we should do to start the ball rolling. A plan of action is what we need. They say that two heads are better than one. Let get a plan going! You can email me or post.

Sandra