This year we're not boasting exciting stories or funny anticdotes. It's time to get serious, we need action.
As you can see from the pictures, Jonah loves: his parents, parties, Pokemon, PJ Masks, racing and friends. Jonah could die in his sleep tonight.
JJB's main focus this year has been on finding more patients. Athough this is our greatest need at the moment, our fundraising efforts suffered because of it. I think sometimes people forget, for the most part, JJB is a two woman operation.
Just a reminder... My husband, Jeremy and I founded JJB when there was little to no research on Sanfilippo type C. We have worked so hard, sleeping little, crying alot, and trying to enjoy every day we have with Jonah. We have a treatment now, it must still go through clinical trial and be approved by the FDA. Our gene therapy drug has cured mice, our drug is on the verge of going to clinical trial. A treatment for our children suffering from Sanfilippo type C is within reach. But the clock is ticking everyday without treatment is a toll on our childrens health.
There is a lot of talk about thoughts and prayers, and although they are very much appreciated we need actions as well. Likes on facebook do not equal funding for research and development. So we ask, please give what you can.
JJB is actively looking for a partner to help us fund our clinical trial and commercialize our gene therapy, but the reality is, that our disease is so rare that potential partners don't see the financial return. So we have to plan for the event that we will have to fund a clinical trial on our own. Every dollar adds up. JJB is proof of that, because of generous donations from people like you we have brought a disease with no hope 7 years ago to the brink of a clinical trial today.