An Austin 13-year-old is determined to find a cure for a rare & fatal disease in order to save his sister’s life. And University of Texas quarterback Sam Ehlinger is helping him. Watch the story with Amanda Salinas HERE!
In May BBDF had its first meeting with the FDA to get input and direction on our plan for clinical trials. This meeting was a huge milestone and an important first step in the process to develop BBDF’s drug combination for the treatment of juvenile Batten disease. (Pictured above: key opinion leaders and BBDF team at the FDA)
We are happy to report that the FDA gave very positive feedback and it was clear they want to help us reach our goal. To put it into context, our regulatory consultants reported that they had never attended a meeting where the FDA was so interactive, motivated and engaged. The FDA guidelines provide a clear path forward for our program and do include some action items for us to address which will help set us up for success when we submit the final package. BBDF, our consultants and advisors are rolling up our sleeves and getting to work in order to get these Batten kids treated as quickly as possible!
Only 6 months until we wrap up our first ever capital campaign to fund the treatment for juvenile Batten disease. Time is of the essence as we still need almost $2 million to reach our $6 million dollar goal but with fundraisers on the calendar and help from our generous supporters we know we can do it! Take a look at our June highlights from last month’s newsletter! READ MORE
2018 has been filled with fundraising, partnership and advocacy here at BBDF. With that said we wanted to share some of the media coverage for the foundation and our family partners around the country!
In it’s 4th year, the annual fall fundraiser, Family Night, is set for Sunday, September 23, 2018 and BBDF is excited to announce that this year’s event will be co-hosted by the Louise H. Batz Patient Safety Foundation based in San Antonio, TX. If you haven’t attended Family Night in the past, we have made quite a few changes and cannot wait for you to join us in “Splashin’ for a Passion!” For more information, including sponsorship packages and ticket information please visit beyondbatten.org/events/family-night.
It’s true what they say, “What starts here changes the world.” Garland has enlisted the help of hometown hero and University of Texas quarterback, Sam Ehlinger, to reach more friends so that he can help fund a treatment that could save his sister’s life! Hook ‘Em!
You can make a donation and stay up to date with Garland’s progress at beahero.beyondbatten.org OR you can text HERO to 501501 and $10 will be added to your next phone bill.
BBDF Welcomes Chase the Cure to our family of donor advised funds. By pooling resources and connecting communities we are able to expand our impact and reach to accelerate research. We are so grateful to have them join forces so that we can one day eradicate juvenile Batten disease for Chase and the hundreds of other children who are affected.
Together Chase the Cure and Beyond Batten Disease Foundation are spreading awareness and raising funds for medical research to eradicate this disease. To find out more about Chase the Cure and the Peterson family visit the website.
Spearheading a unique, cohesive strategy, Beyond Batten Disease Foundation has added ThinkGenetic to its collaborators by joining the ThinkGenetic Advocacy Partner Program.
“Partnership and collaboration have been the cornerstone of Beyond Batten Disease Foundation’s success and we are excited to work with ThinkGenetic, ” said Mary Beth Kiser, President and CEO of Beyond Batten Disease Foundation. “As a rare disease patient advocacy group, we appreciate the importance of creating awareness and sharing information. Beyond Batten has a robust research portfolio and a principal scientist on staff. We hope to share information and serve as a resource to juvenile Batten patients and beyond. ThinkGenetic is the perfect platform to help us do that.”
Soon after the first-ever therapy for Batten disease was approved by the U.S. Food and Drug Administration (FDA) in April 2017, Danielle Kerkovich, principal scientist at the Beyond Batten Disease Foundation, started fielding questions from families wondering if their child could take the new treatment.
Kerkovich had to tell parents that they, unfortunately, could not. That’s because the Beyond Batten Disease Foundation works with kids who have the CLN3 form of Batten disease. Brineura (cerliponase alpha), developed by the pharmaceutical company BioMarin, can’t be used for CLN3 — it was designed for patients with the CLN2 form of the disease.