This year, our 30th Anniversary, a monumental stride was made for the Batten disease community. Brineura™, the first-ever FDA and European Commission approved treatment, became commercially available. This treatment took years of commitment from donors who funded the building blocks that made the clinical trial possible. Thanks to your support, as a Patient Advocacy organization, we were equipped to get actively involved with the process – a move which ultimately tipped the scales of receiving final approval.
The FDA and BioMarin both told us that without mobilized patient voices at the table in the drug development process, there would not have been an approved treatment. We would be back to the drawing board. The FDA recognized that there might have been more benefits for children than what was showing up in the statistics. BDSRA’s families going to the FDA made all the difference, and now children all over the country are being treated. ” – Margie Frazier, BDSRA Executive Director
We are energized by this and yet are acutely aware of how much more there is to do for all forms of the disease. It truly takes a long-term commitment to support, advocate, and build a better future for children and families impacted by Batten disease.
In 2018, we are taking a global look at what we have and what we need with regard to science and research. What is in the gap? What are the global needs of the research community? How can funds be targeted to achieve the next steps? While we strive for research to impact the future, we know that support services are the lifeline families rely on today. We continue to stand in the gap for families every day – supporting those who’ve received this diagnosis.
Meet Nathan Hiltman, a vibrant, energetic boy diagnosed with CLN3 on June 10, 2015. Like most of his peers, Nathan loves Star Wars, Disneyworld, and Pokémon. Nathan is a very social kid who loves going out to dinner and to the mall with his family.
Sadly, just this year he has started refusing to go anywhere because he is “afraid he is going to fall.” Nathan’s mother, Stacey, has sought out guidance and support from BDSRA and our Facebook community with other parents going through similar experiences. Some of the support services the Hiltman family received included one-on-one education training and materials, equipment exchange, and the family conference. These services are here for a reason, and we thank you, our supporters, for making this possible.
BDSRA is the place where families turn when they need their voices heard. We are the place where they feel understood and safe. We are always calling, writing, traveling, thinking, and speaking as their Advocate. It is our job to work on behalf of all children and families. And it is our honor to seek out and disseminate all we can about the global landscape for Batten disease.
While we celebrate 30 years and the first-ever FDA approval, we know these milestones do not mean our work is complete. We are excited to build on this momentum and create new possibilities in the lives of the children we serve.
We see this as a mark to honor what we’ve done and plan for the next 30 years. We have a legacy to create for all of the lives that have touched our hearts and for all of the faces we have yet to meet. They deserve the best we have, and we are so grateful for your financial support to ensure we can be here to deliver it. Individual donors make up the majority of our funding.
If trends continue, we will see more families than ever next year. We are also anticipating gene therapy clinical trials in 2018 and new researchers on the scene expanding the community’s library of knowledge in basic and translational science.
These are the very building blocks that made our first FDA approval possible and we are committed to continuing the work with your support.
Please consider a generous gift to our 30th Anniversary Annual Fund, so BDSRA can continue to represent the patient voice across the country and across the world.
Board President, BDSRA