One year ago almost exactly, our son Joseph was diagnosed with CLN7 – one of the many terminal forms of Batten disease. In the year that followed we have learned more about genetics, rare disease, community, support, non-profit foundations, fundraising, web site development, social media, friendship, Texas, kindness, family and faith than we ever could have imagined learning in a lifetime.The past 12 months have been the best, the hardest, and the most inspiring of our lives. With the help of our friends, family, and community, we were able to do the impossible, and raise the funds we needed to start the dose development for the clinical trial – a milestone that so many worked so hard to make a reality.
Thank you so very much, for making it an incredible and miraculous success in such a short amount of time.
As we look to 2018 there are new challenges ahead but still the promise of hope and miracles runs strong. For our family and the CLN7 clinical trial, we still have a large amount of fundraising to do in order to get the pre-trial work completed. Jojo is changing everyday in response to Batten disease. Today he has lost much of the sight he had one year ago, his walking is now cautious and less steady, but he is still telling stories, he is still working puzzles, still antagonizing his little sister, and still jumping on his trampoline. He is beyond excited to go to school with other children in the next few weeks, and he will eagerly tell you that his sixth birthday is just around the corner.
We have also met new families facing a gene therapy journey all their own, some facing CLN7 with us, and others confronting other diseases that are uniquely difficult and in urgent need of gene therapy. As a foundation, our goal is to help them realize the same experience of hope and possibility that we were so graced with in the past year. We are excited to introduce you to these families and to the growing gene therapy program that will change the world, starting right here in Dallas. The people in this journey are miracle makers – it is impossible not to fall in love with them and their love of life, and it is inevitable that we be inspired by their strength and commitment to changing rare disease for the better.
As we look toward the milestones of 2018 one thing is abundantly clear, and that is the hope we continue to have for Joseph, and for so many other beautiful girls and boys given a chance to change their terminal diagnosis through life-saving gene therapy. We are so grateful to be a part of this journey with them, and with you. Everyday is truly an awesome day.