The BDSRA Foundation officially announced Boys Town National Research Hospital as the newest site in the Batten Disease Clinical Center of Excellence Program and the promotion of NYU Langone Health and UNC Hospitals from “Affiliate Centers” to “Centers of Excellence.”

On February 27, you can order from Panda Express online to support the BDSRA Foundation and families affected by Batten disease. We raised over $2,300 last year, and this year, we challenge you to spread the word to help surpass last year’s proceeds! 

Right now, you can support the BDSRA Foundation by purchasing some sweets from See’s Candies! Buy some assorted chocolates before Valentine’s Day, or snag some Easter candy to get a head start on those Easter baskets! Find all that and more and help benefit the Batten disease community by clicking below. 

Want to plan a birthday fundraiser for BDSRA? We are here to help!

BDSRA can create a custom link and QR code for you to use for a birthday fundraiser that directs donations to us! Creating a Fam Fund is another great way to raise funds to celebrate your birthday, and we can do all the legwork for you. 

If you are interested in setting up a Fam Fund for a birthday fundraiser or a stand-alone birthday fundraiser this year, please email Heather Dainiak at heather@BDSRAfoundation.org to get started.

We are proud to share a major new review on the natural history of CLN3 disease, led and driven by members of the BDSRA Foundation’s Batten Disease Clinical Centers of Excellence (CoE), the Batten Disease Clinical Research Consortium (BDCRC), and esteemed international partners.

This important and practical meta-analysis brings together data from more than 400 individuals, integrating over two decades of natural history research to define a clearer timeline of disease progression in CLN3 disease. The findings provide valuable anticipatory guidance for clinicians and families and may contribute to improved clinical trial design and therapeutic development.

This work reflects the strength and impact of sustained international collaboration and the exceptional dedication of our global research community. We are deeply grateful to — and proud of — our CoE, BDCRC, and international collaborators for their leadership and long-standing commitment to improving outcomes for families affected by Batten disease.

Congratulations to the outstanding author team led by BDSRA’s Dr Ineka Whiteman and University of Rochester’s Dr. Heather Adams, with Anthony (Tony) Cook, Erika Fullwood Augustine, Aidan D. Bindoff, Alexandra M. Johnson, Heather Manson, Jonathan W. Mink, John R. Østergaard, Angela Schulz, Jennifer Vermillion, and Amy Vierhile.

“CLN3 mediates chloride efflux from lysosomes”

“Enteric nervous system degeneration in human and murine CLN3 disease, is ameliorated by gene therapy in mice”

“Limited therapeutic efficacy of N-acetyl-L-leucine in a mouse model of CLN1 disease”

The quarterly research column written by our Head of Research & Medical Affairs, Dr. Ineka Whiteman, will be published in the February edition of The Illuminator in early March. 

After a two-year campaign to reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program, the rare disease community’s relentless advocacy has paid off!

Click below to read the full summary from the EveryLife Foundation for Rare Diseases.

The BDSRA Foundation Family Register is a vital tool that enables us to keep you informed of ongoing Batten disease research, including future clinical research and natural history opportunities.

The information collected in this form is kept STRICTLY CONFIDENTIAL. Your involvement in this survey is entirely voluntary, and you may request to be removed from the list at any time. The form takes just a few minutes to complete and can be accessed by clicking the button below.

Together, we remember those we have lost to Batten disease. We work daily to build a brighter future for families in their honor and memory.

In this newsletter’s edition, we honor and remember:

Seth Wyatt Cobb

Breanna Lee Neveri

To have your loved one’s name placed in The Illuminator and read at the Celebration of Life at the Annual Family Conference, please email patrick@bdsrafoundation.org.

Thank you for investing in our mission. We exist to provide support, research, and advocacy for families with all forms of Batten disease. We want to acknowledge the donors who gave so generously last month to our work.

Together, we are Batten Advocates for a Cure.

To view the list of recent donors, please click the button below!

From copy paper to personnel, $5 Fridays is designed to keep the office moving. The more $5 Friday donations BDSRA Foundation receives for operations, the more money BDSRA can invest in programs, research, and advocacy.

That’s the power of FIVE. 

We connect your donation to Support, Research, and Advocacy for families of all CLN types.