With only four months to go BBDF and our Batten families are hard at work to fund the clinical trial! Take a moment to see what our next steps are and catch up with all of the activities of our affected families in our August Newsletter! READ MORE
by JAMIE KIM
My brother, Tyler, has always been the most energetic happy little boy I have ever been around. He never runs out of things to do or things to say.
A few years ago, he started to have seizures and began to lose his vision. Around a year ago, our family found out that Tyler has juvenile Batten disease. Batten disease is a rare and awful neurodegenerative disease that is terminal. There is currently no treatment or cure but there are researchers who have discovered a treatment that slows the progression of the disease and others are working on a possible cure for the disease.
When I first found out he had this disease, I was a little in disbelief. I have seen many commercials of people diagnosed with terrible diseases but it never occured to me that it could happen to Tyler. He seemed like such a happy and healthy boy that it was hard to understand that such a devastating disease was hurting him.
In the first few months of his diagnosis, I did not want to talk about it with anyone outside our family. I did not know how to tell people what he was going through because it is not a common disease and I knew no one would really understand what was happening. Our family began first with telling close friends and extended family. More recently, we joined other affected families and set up a fund at Beyond Batten Disease Foundation called Tyler’s Mission. All donations to Tyler’s Mission go to the research for a treatment and cure for juvenile Batten disease. Seeing all the people who have donated to this cause has showed me how many people care and support our family and it really means so much to us. It did not cross my mind that so many people who do not know us were also donating and leaving comments of support. Looking at these comments made me realize that it is possible to raise the 6 million dollars needed by next year to start a clinical trial for the treatment for kids living with Batten Disease. So far, Tyler’s Mission has raised over $45,0000!
This summer, I want to make a difference. I want to help raise money and awareness of Batten Disease. It is urgent to raise the funds as quickly as possible because time is not on our side. With donations and support, there is hope for a treatment and ultimately a cure.
Every day I am reminded there is hope for my little brother and others with Batten Disease. I love Tyler more than words can explain and he brings me happiness everyday from his little kisses to his big squeeze hugs to his goofy bad jokes. Although he has hard days and struggles sometimes, he always hold his head up high and helps me see how much there is to appreciate in our lives.
Please help us raise funds and defeat Batten Disease by donating to Tyler’s Mission at beyondbatten.org/tylers-mission or visit the Understanding Batten page to learn more about Batten disease. To stay updated with his journey, follow his Instagram, Twitter, and Facebook @tylersmission.
GO TYLER! BEAT BATTEN!
Long time BBDF supporter, Joe Sikorra has published a book and dedicated a portion of the proceeds to BBDF! You can snag a copy via Amazon, but remember to shop via Amazon Smiles (www.smile.amazon.com) so that .05% of your purchase will be donated to your favorite charity. You can purchase the book directly HERE.
This moving story shows how a family found joy after their hopes and dreams were shattered by the rare, fatal neurological illness of their two sons. Told by the family’s father, a popular Catholic radio host and marriage counselor, it recounts the way he and his wife received the courage and the support they needed to give their children abundant, fulfilling lives in spite of their gradual decline.
What initially seemed to be a tragedy for Joe Sikorra’s family became a story of human triumph, an outcome made possible by the compassion and mercy of God. Their example can help those facing unexpected losses and challenges to believe that by placing their trust in Providence they too can overcome hardships with the most powerful force on earth—God’s love.
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!
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.
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.