We know that making the decision to participate in a natural history study is not a small one. Taking time away from an already challenging rare disease life is a lot to ask. As the parents of one of the sweetest little boys facing Batten CLN7 disease, here is our reasoning for why we believe the joining a natural history study is worth considering.
Reason #1. You are here, and you can help.
I remember what life was like with Joseph before we knew Batten disease existed. Back then, our little buddy could run, wave at a passing train, and ask for cheeseburgers for dinner like he didn’t just have one for lunch. If we could have never entered into the rare disease world our family would be living a very different life today.
But as of today, a treatment for Batten CLN7 has not yet been built, and we are facing this rare disease in real life. The hope for us comes with the realization that Joseph is uniquely able to make a big difference, and regardless of the outcome of our work toward a clinical trial, anything that Joseph can do to help resolve CLN7 for him, and for other families is worth doing. The knowledge from the natural history study matters. Whether that benefit is in the form of a treatment, or just an understanding of the progression of the disease the work must be done.
We feel strongly that by making his experience in this rare disease a part of the research that benefits future children, he is beating CLN7. Period.
Reason #2. Participation may actually help, today.
When Joseph was diagnosed we had a hundred questions about time, progression, support and treatment options. We were amazed at how limited the answers were.
We are excited to get Jojo some time with Batten disease experts in the natural history study – it’s another opportunity to ask questions, learn from other families with his disease, and expand our own understanding of Batten disease.
For us, every bit of insight into additional information that may help Joseph is motivation for us. We want all the help we can get to navigate his disease, to support others in their journey, and we hope that our participation in the natural history study will help to build a treatment that will help to save his life.
Reason #3. It takes a village…let’s build one for Batten CLN7
With three other children, jobs, and Joseph’s unique challenges, we know how difficult it is to take time away for a even a few hours. The natural history study takes days away from your normal life, and a that’s a lot of commitment. But we are here, in the world of rare disease, and we are committed to changing the future for families facing Batten disease.
You don’t have to do it alone. A natural history study is typically sponsored by a research organization, and this is true for CLN7. This means that the expenses to participate are often covered, so you don’t have to worry about the financial burden of participation. Instead, you can focus on making it a positive experience for your child, learning, and moving forward in the development of a solution for your child’s disease.
Every family has to make their own decisions, and the right one for your family really is the best decision you can make. If you can join the study, I hope you do. If you can support the study remotely I hope you do. For our family, we believe that by coming together as a community in support of the knowledge needed to build a treatment for Batten CLN7, we are one step closer to building the village that the children of Batten disease need today.
The CLN7 and CLN5 natural history study begins in February of 2019. If you are interested in joining us, here are some FAQ’s that might help you in your decision. You can also review the study publication at clinical trial.gov or reach out to the call center at 1-877-237-5020
