My head continues to spin. I was fortunate enough to be able to attend the Beyond Batten Foundation’s 1st ever in-person meeting on the state of research for CLN3. Families from many states (CO, D.C, MA, MI, NE, OR, PA, TX, UT) and even the Netherlands were represented. BBDF founders Crag and Charlotte Benson and HOPE founders Wayne and Missy Herndon along with BBDF President and CEO Mary Beth Kiser held a dinner for the family partners on wednesday night. This was my 1st time in contact with other families that are dealing with the realities of Batten, some for a year, some for 26 years! I felt an immediate calming. These strangers got it, they understood what my family was going through; and the families with older children really lifted me up, showing by example, that we can continue to live, and to love in spite of this condition.
On thursday we got to tour Texas Children’s Hospital Neurological Research Institute. A massive 13 story research facility that was developed from the ground up specifically to study Neurological disease, including Batten. We got to see the research lab where a team lead by Assistant Professor Dr. Marco Sardiello has been studying treatments for juvenile-batten. We were then briefed on the current medical state by the NIH, Vandenberghe Lab, Abeona, Amytrx, and Dr. Sardiello Ph.D and Dr. Gary Clark M.D. There was a tremendous wealth of information provided. I found the general mood of the professionals who have dedicated their life’s work to curing Battens to be energetic and exciting. Dr. Clark said this is the “most remarkable time to be in pediatric neurology. We are where pediatric cancer was 20 years ago.” For a disease that was identified in the 1820’s, for the first time ever, medical professionals have real tangible hopes for several treatments on the very near horizon. We plan to carefully weigh our options and reach out as the above medical organizations begin their clinical trials.
As a direct note to you, our goFundMe village: I believe we all work hard for our money. I also believe you are our partners in this journey. Some of you we know, some we have never met, but I want you all to know how much your support has and continues to comfort us. It was so incredible to be able to come to this conference, and moreover not to have to worry about the cost of lost work and travel expense to attend. This is an incredible gift; thank you! This journey is so unexpected, but together we will make it.