My first blog....On the road again.
Jeremy and I are heading to a "Sanfilippo Workshop" in Northampton. The workshop is being hosted by the UK MPS society, it's goal is to help bridge the gap between: pharma, research and Sanfilippo advocate groups. We're very excited to be a part of this meeting for several reasons. They are as follows:
Our researchers Bigger and Pshezhetsky will be in attendance. Pshezhetsky is working on a mutation specific therapy for Jonah and friends. JJB hopes to fund Bigger on a viral vector ASAP. (I just can't get enough of our researchers. I wish I could sit in their office's and work, so I could watch the lab techs work on a cure for our kids.)
Brett Crawford from Zacharon will be attending. The Sanfilippo community is very anxious to learn more about the potential trial that Zacharon is working on in partnership with Pfizer. Pfizer just gave Zacharon a $210 million dollar contract to help take their "small molecule" to clinical trial. At this point this potential treatment is Jonah's closest hope.
