A world where there is no PMD: How to get there through Research
Friday and Saturday, April 17-18, 2009 -- Alfred I duPont Hospital for Children Wilmington, DE
Marcy and I are happy to sponsor the second PMD Symposium mid next month. Of course we will be attending as we did five years ago when we first learned of the horrible disease that plagues our Jacob. We met so many valuable US based medical researchers who have helped us with our ongoing research efforts to find a treatment for Jacob and other boys like him.
While our first two experimental investments have not yet produced treatments, we believe we're getting closer and closer. In fact, we're already in negotiations for our next investment with a speaker who will present some recent work at the symposium next month. Expect to hear more about this soon. We are very excited about the potential for near term treatment opportunities with this next round of experimentation. Some experimentation using off-lable medication with animal models with PLP duplications has already successfully shown down regulation of PLP expression along with verifiable and quantifiable clinical improvements. We hope to show that similar benefits can be had with severe point mutations like Jacob's.
This is extremely exciting and we can barely contain ourselves. But as you probably know, medical research is both extremely expensive and unbelievably time consuming. Your donations are greatly appreciated and going directly to fund very important research that could help Jacob and other PMD kids like him very soon. We'll keep you posted.