Archive for 2 March 2010
2 March 2010
The last time we wrote an update, we already mentioned that Jasper is regressing. since then, things have certainly not improved; Jasper really has difficulties with walking so we have had to start focusing on other activities. He is clearly frustrated about it himself and his behavior reflects his sorrow. We all know it’s part of the disease, but it remains difficult for us to be so harshly confronted with the progression of the disease.
Seizure control is fine; we found a good balance in the new seizure meds. Eating and drinking is also still great!
Because Jasper has become a big 5 years old kid (who weighs about 45 lbs), we really started to struggle with getting him in and out of our car (in his special seat). Therefore we had to trade in our Volvo station wagon for a large SUV (Mazda). Now we have much more room to put him into the car and we don’t have to bend over so much. And since we have a huge trunk, there is also no longer a need to collapse his special need stroller. So, we really did our backs a favor here!
Fundraising is still going very well. Last weekend, we had a poker tournament in Amsterdam for exchange traders. The total proceeds were about $65,000. That was considerably more than we hoped for. A big thank you to the Amsterdam trading community!! In the mean time, we have raised more than $900k in funds for LINCL research since we started this adventure in May of last year. Of course, we also started spending the money to fund various research projects. We aim to get to the $1 million mark within our first year of fundraising. A bit less than 2 months to go…
We initially started this project last year to co-fund the clinical gene therapy trial at Cornell University in NYC. At the time, this was the most promising (and only available) therapy that was ready for a clinical trial but there was no funding available. However, recently things changed in Cornell’s favor and they received funding from the NIH to execute their study. This is good news for us as it means that we can start funding other promising research as well. Cornell will start the screening process of potential participants shortly but since it looks like the eligibility criteria have been tweaked, it remains to be seen if Jasper will still qualify to participate in the study…
On the research topic; I also spend a couple of days down in Miami last month to attend a Lysosomal Storage Disease conference. It was great to catch up with some people and meet other researchers in an informal setting to learn about the latest in the world of science.