It's Thursday night and I'm winding down and getting ready to hit the sack. I have to work Friday and Saturday. I'm spending Sunday packing and mentally preparing myself for what may be a big step forward in dealing with my disease.
On Monday morning, I'm flying to the National Institute of Health in Bethesda, MD. I will be participating in a new study that will hopefully lead to a new treatment for Late Onset Tay-Sachs disease. This is the beginning of the first step of the study. This is pretty much data collection that will be used to develop outcome measures that are needed in order for the FDA to approve any kind of treatment.
This stage of the study will last about six months I think. The other ten or eleven participants and myself will be wearing data collection devices that will relay data back to the researchers. When this portion is done, they will hopefully have the treatment ready to test. It is pretty scary, because it is currently in animal testing. If that goes well, we will be the first people to try this treatment. It is going to be gene replacement therapy, that will hopefully at least slow the progression of the disease, if not reverse it.
As I said earlier, this is pretty scary. But somebody has to do it if we ever hope to find a cure. I am willing to give it a shot. It may be too late for me to be cured, but I'm hoping that the data I provide might help someone down the road in dealing with this horrible disease.
I'll be bringing my laptop with me and I'll post some updates while I'm in Bethesda.
Take care all, and have a great weekend!