NOTE: There won’t be a December edition of The Illuminator. We will resume the newsletter with the January 2025 edition in late January or early February. 

Please check out our office hours for the holiday season. We will also be closed on January 1st and return to regular office hours on January 2.

Last month, we announced our **$150,000 fundraising goal** for the 2024 Annual Appeal!

Every dollar raised will support Batten disease families through programs like the Annual Family Conference and Grief Chats, fund and facilitate research, and help our community advocate for treatments and a cure. We look forward to taking another step toward our vision of a world without Batten disease!

What’s a BDSRA project, initiative, or cause you’re passionate about?

Giving Tuesday may be over, but there’s still time to raise funds for whatever you’re passionate about through the end of 2024.

Here’s how YOU can help raise funds for a BDSRA project, initiative, or cause that resonates with you (click the button below for full details):

1. Share a letter with your community

2. Post on social media

Read our 2023 highlights in the 2023 BDSRA Impact Report! The report includes stories on Support, Research, Advocacy, and Fundraising as well as letters from the President & CEO, and Board Chair.

Click the button below to read.

It’s time for research updates! BDSRA Foundation’s Head of Research & Medical Affairs Dr. Ineka Whiteman breaks down Batten disease clinical program updates, research news & opportunities, and resources in her monthly column.

Her latest column includes:

• NCL Translational Research Conference summary
• Clinical program updates
• Write-ups on the latest Centers of Excellence meeting and her recent visit to RUSH University Medical Center
• Publication summaries

All good things must come to an end. So, it is with the BDSRA Foundation Board of Directors that we say thank you and goodbye to two long-time board members Donna Fogle and Tony Ferrandino, who’ll leave the board at the end of this year. They are both bereaved Batten parents who have channeled their grief into helping others.

Tony lost his younger son Drew and Donna lost her only daughter Kourtney to Batten disease. Years have passed since they left us, but the need to help other families on this painful journey inspired them to continue the fight by joining the Board.

Donna’s background is in healthcare insurance. She has used her knowledge and passion to campaign for newborn screening in Florida and to meet routinely with the Florida delegation in Congress. Whenever she goes to meet with legislators, she carries a picture of Kourtney with her to channel the discussion to rare disease legislation.

Tony is a financial guy. He worked for years at Merrill Lynch and in that capacity provided advice and counsel on the restricted and unrestricted funds in BDSRA’s portfolio. He has also provided advice to Batten parents as to how to set up funds to provide for their affected children.

We have appreciated their knowledge and expertise all these years. We will miss their presence at board meetings and the sage advice they provided. Most of all, we will miss their smiles and hugs.

There will always be a seat at the table for both of you.

-Contributed by Barbara Wuebbels, Board Vice Chair

The Translational Research Conference for the Management of NCLs was held November 20-22, 2024, in Chicago, and many members of our Batten community attended, including BDSRA team members Heather Dainiak (also Conference Co-Host), Dr. Ineka Whiteman, and Amy Fenton Parker. Many parents were also in attendance, and one first-time attendee wanted to maximize the opportunity to advocate while there.

Naperville (Chicago suburb) is home to Matthew and Leigh Sims, who are the parents of two sons. Their younger son, Charlie, is living with CLN1. Last year, Charlie became the first human ever dosed with a gene therapy for CLN1, which was delivered into his spine and brain at BDSRA’s Center of Excellence Affiliate RUSH University Medical Center by Dr. Liz Berry-Kravis and Research Director Kendall Robbins. Both were also in attendance at the conference, along with other Chicago Batten parents, Tracy and Jen VanHoutan and Dave and Sara Kennicott.

Because so many people from the Batten community were gathered in one space, Sims contacted his Congressional Representative Bill Foster, PhD, for a meeting. Foster serves on several committees and caucuses, but most importantly, on the Rare Disease Caucus. He agreed to meet with the group to learn more about Batten disease and the needs sought to be approved before the year’s end. When the conference concluded at 5:00 PM on Friday, the meeting began.

Congressman Foster spent an hour with Matt Sims, Tracy VanHoutan, Berry-Kravis, Robbins, Fenton Parker, Whiteman, and Jill Weimer (Conference Co-Host) of the Weimer Lab at Sanford Research Center. The group discussed Batten disease, the biotech downturn, and the FDA approval process, along with the effect it has had on treatments and cures for the Batten community.

The group also discussed bills currently in consideration for voting before this session ends. Most importantly, the group urged the congressman to vote for the Accelerating Kids Access to Care Act, the MVP Act (Medicaid Value-Based Payment Arrangements for Patients), and the Rare Pediatric Disease Priority Review Voucher Program Reauthorization.

BDSRA makes it a practice to maximize time spent at conferences like these, including the Centers of Excellence meeting the day before the conference began. It was a welcome opportunity to end the conference by advocating for the families, the research, and the clinical care that was shared all week.

Click below to read the letters BDSRA signed on to during October and November:

• Letter in support of an epilepsy objective being included in Healthy People 2030
• End of Year Letter to Congressional Leaders on 3 Rare Disease Bills
• FY25 Epilepsy Appropriations Organizational Support Letter
• Community Support Letter for the Rare Pediatric Disease PRV Program

“Uplifting Experiences can take on many different forms, but the top priority is always harnessing the power of sport to inspire hope and joy in the lives of people impacted by rare diseases.

The objective of each event is to provide an opportunity for members of the local rare disease community and athletes to connect. Uplifting Experiences is centered on affording people impacted by rare diseases a unique and memorable experience, while simultaneously allowing athletes to directly connect with the people impacted by a cause they support.”

Click the button below to view the Uplifting Experiences upcoming events list to learn more and register.

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.

The holiday season can be a difficult time for bereaved families. As part of our Life Goes On program for bereaved Batten parents, BDSRA will hold its next virtual Grief Chat on Sunday, December 15 at 7 p.m. EST.

Grief during the holiday season will be the focus of this session. If you’ve already completed the Grief Chat form, you will receive multiple email reminders before the chat.

These Grief Chats are an opportunity for bereaved Batten parents to gather and support each other no matter where they are on their grief journey.

If you haven’t attended a Grief Chat this year, please click the button below to register.

Want to see more content from BDSRA? Don’t miss out on upcoming events, information, campaigns, and community posts! Follow BDSRA on Facebook, Instagram, LinkedIn, X, and YouTube to stay in the know on all things happening in our Batten Community!

Click below to follow all of our accounts: 

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.

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.

Fisher Aslin

Lillian G. Kerl Jackson

Gabriel Lindemeyer

Grant Peter Severance

Brianna Swindall

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.

BDSRA is grateful for all gifts that support the mission; however, restricted gifts require more time and attention to administer. At its July 11, 2024, meeting, the BDSRA Foundation Board of Directors voted to implement a management fee of 7% on restricted gifts effective August 1, 2024. Please contact President & CEO Amy Fenton Parker with any questions at amy@bdsrafoundation.org.

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. 

Designed to give our community control over their stories, fundraising, and donations, our Fam Funds participation is growing!

• Tell your story
• Raise funds for BDSRA
• Choose where donations are directed
• Share with your community

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