Marilee's Latest Post on the condition of her boys. . . . .

I find it phenomenal  that Marilee has TWO children with the same disease and they
are the ONLY ones in the entire state of Arizona that have it.

Adam and I debate regularly how many children in the world have the
same cancer as Anthony: Acute Lymphoblastic Leukemia on the B-cell.
Adam thinks this is extremely rare and anything that indicated the
above cancer geographically would violate their rights to privacy.


Sept. 2 - Appt. with Biochemical Geneticist - Today I drove Eli and Evan to Phoenix for an appointment with Dr. Alec. (Brad was going to come with me, but he ended up having to go to work, so I was on my own.) Dr. Alec is the biochemical geneticist who diagnosed the boys one year ago. Dr. Alec has become more and more interested in researching and treating children with different enzyme disorders by Direct infusion into the brain (probably through spinal tap). So I told him about the research that is being done right now with GM1. And how they're hoping to have clinical trials within the next few years. He hopes that if they do figure out the technology that it could implemented here in Phoenix to treat children. He believes that Eli and Evan are the only ones in Arizona that he knows about with the disorder. He hasn't heard of anyone else in Arizona and he thinks he probably would have. But it's possible there might be more. I gave him an update on the boys and gave him a more full history about Evan since he never got that from us. I told him about Minnesota, at least what I could remember. And about the clinical trial with miglustat. He said it seems as if we have been looking into every possible road and encouraged me to reach out to NIH if I felt so inclined. He told me he would stay updated on any of the latest with the research going on and GM1 and contact me if he hears of anything. He will also try to sit down and decide if he thinks they're more close to the late infantile or the juvenile type. Not that it will make any difference or even really tell us anything. But might be helpful to know. It was a long day of driving and a long appointments, so the boys weren't too happy. But we got through it and it was good and I'm glad that we were able to go. The boys after our appointment in Phoenix, Eli had no pants on because I had to change his diaper during the middle of the appt.! Both had incomplete foot wear by the end and were very tired!   

Marilee Burdetts


This is a link to Marilee's blog that I took this from. I am too tired to try to change
it so it doesn't look like this.