FDA Meeting Update


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!

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June Update – Only 6 months to go!


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

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BBDF and the Louise H. Batz Foundation bring you Family Night 2018!

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.

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Calling all Heroes!

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.

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BBDF Welcomes Chase the Cure

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.

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Batten Families Gather at TCH for Research Update

For the first time in foundation history, affected families from all over the world gathered at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital. The families were able to meet the research team, tour the lab and learn more about BBDF projects. Additionally, the foundation brought in outside experts and researchers to share information about other treatment options on the horizon.
“It meant the world to me to be surrounded by such positive and motivated people. I am just in awe of Beyond Batten and all of your collaborators. You have given us such real hope.” Family Attendee
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BBDF Partners with ThinkGenetic in Support of Mission

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.”

READ FULL ARTICLE HERE

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Brineura Approval Invigorates Research, Awareness About Other Batten Treatments

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.

CLICK HERE TO READ THE FULL ARTICLE

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Over $70,000 raised in February!

It is an exciting time for the Foundation and our affected families as we near the possibility of a clinical trial for the first-ever treatment for juvenile Batten disease. Our February newsletter highlights a new donor advised fund, Bind Up Batten, three community fundraisers, a plethora of ways to get involved and of course your monthly update on the Be Project, which is now up to $3.7 million dollars towards our $6 million goal! You can read the full newsletter HERE!

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