View sessions from the 2025 Annual Family Conference in Lincoln, Nebraska, by clicking the button below!

DON’T FORGET: Join us in Chicago for the 2026 Conference from July 10-12

Dear Batten Community,

We are pleased to present the Annual Family Conference session videos for everyone. Maybe you couldn’t attend the conference, maybe you wanted to be in two places at the same time, or maybe you want to review what you saw while you were in Nebraska. We hope you enjoy this investment made by BDSRA and the conference sponsors. Please thank them for their generous support.

I also want to thank everyone who attended this year’s Annual Family Conference in Lincoln, Nebraska. The local families who helped us were fantastic, and we could not have made it happen without everyone. “It takes a village to care for a village.” This is my new motto.

It is so helpful to have our families complete the conference evaluation surveys. We studied your suggestions to help us plan for the next year, and I wanted to share a few things with everyone in response to the suggestions we got this year.

1. When we look for a location for the conference, we work hard to meet the needs of all who attend. We give our event planning company a list of places we would like to hold a conference, and they send out requests for proposals with an extensive list of requirements. First, we look for hotels that will not cost more than $170.00 per night. We also look for an airport nearby that has international flights, but that is not always easy, and most families drive. We look at the number and size of the elevators, as well as the size of the lobby. We must have pool, accessible parking, and enough meeting rooms for all our sessions. We consider the cost of food and beverages too. When we pick the city, we think it would work best for us, we go check out the hotel. In May, we visited four different hotels in the Chicago area before we chose The Westin Lombard. Too often the price of a room is too high for our conference, especially on the east or west coast. We have found that the Midwest is more cost-effective, and the attendance is best.
2. We have offered airport transportation reimbursement in the past, but none were requested. We can look for a sponsor to help families with airport-to-hotel travel expenses who have affected children.
3. For the first time in the 20 years that I have helped with the conference, we did very well with the food selection. The Dinner Dance buffet on Saturday night seemed to be well-liked. People were not waiting to be served their dinner, and they could go back for seconds. Giving the children their meals directly at their table, so parents did not have to get their food too, seemed also to be popular.
4. Trying to set up an agenda is never an easy task. Each year we try to produce something we hope is of interest to our families. We know that science and research are always important to everyone, especially anything new. The rest is a gamble, but we do try each year to improve the sessions we offer, thanks to your commitment.
5. The breakout groups are an important part of the conference for most of the attendees. To keep it going, we need moderators to lead them. We have training available, and you could be a co-leader. If you would like to moderate next year, please contact Amy, Heather, or Linda.
6. We have mixed reviews with a keynote speaker, and we have skipped having one, too. We have tried everything from football coaches to Batten moms. Coach Busch was brought to us by the local community, and we accepted the gift of his time. Your survey responses are important to helping us select or go without a speaker, so if you have suggestions, please let us know.
7. One hidden gem is our Explorers’ Camp. We hire a professional group to come, and the Our Promise to Nicholas nurses volunteer. We find sponsors so we can offer free day care for all those affected and children five and under at the conference. I want all the new families who are considering coming to next year’s conference to know this is a safe place to bring your kids while you attend the sessions, and they always have great activities planned. Thanks to the Larson family, we had a lot of extra volunteers this year.
8. Thanks to Bev and George Maxim from Canada back in 2002, we now try to do something special for the bereaved parents who keep coming to conference after they have lost their child. The Life Goes On program welcomes parents in all stages of life coming to the conference, and they are all needed to make up our village. The new and young, those in the middle, strong, and ambitious, and the wise and ageing are all needed, no matter where they are on their journey.
9. We cannot forget our incredible SIBS program which has been around for 35 years now! They help create their own weekend, and we search for activities that everyone will enjoy. We have had dedicated volunteers leading the program for a long time now, and we are in need of some new leaders. If you know someone who might be interested in helping to lead the SIBs program, please let us know.

Enjoy the conference videos, and if you have any trouble, please email Patrick Kotnik, Patrick@BDSRAFoundation.org. We look forward to seeing you in Chicago next July 10-12!

Darlene Royalty

BDSRA Board Chair

A case report from the team at Sydney Children’s Hospital (SCH) in Australia reports on two sisters treated with oral miglustat for 18 months, demonstrating improved visual acuity and clinical stability, with no major side effects. While miglustat is still an investigative therapy for CLN3 disease, this case report is an important step in understanding its potential utility as a treatment. 

Congratulations to the SCH team, including BDSRA Australia’s Medical Advisory Board members Prof. John Grigg, Dr Alex Johnson, and BDSRA’s Ineka Whiteman.

In 2024, recognizing the urgent need to establish research priorities across the basic, translational, and clinical sciences to tackle the most pressing concerns of the Batten disease community, the Batten Disease Clinical Research Consortium (BDCRC) was established. This U.S. academic network is designed to serve as a collaborative framework to advance clinical research in Batten disease. Led by Kennedy Krieger Institute (MD) and the BDSRA Foundation, a Priority Setting Partnership gathered community input to identify top research goals. The results informed the research strategy for the Batten Disease Global Research Initiative (BDGRI) and, excitingly, were just published in The Lancet Neurology journal.

Congratulations to the BDCRC authors and our own BDSRA Foundation team members! 

In recent years, considerable progress has been made in the understanding of Batten disease cell biology, disease pathogenesis, and potential links to other disorders. In this review, Professor Jonathan Cooper and his team at Washington University in St. Louis highlight these advances and how they inform the ongoing development of therapeutic strategies and their future prospects.

Publication of Dr. Ineka Whiteman’s research columns is switching from a monthly to a quarterly schedule. Her next column will be published this December. 

Registrations are still open for this premier Batten disease conference, which unites top researchers, healthcare providers, industry partners, innovators, and patient advocacy leaders globally. Learn more and register by clicking below. 

Click below to read the letters BDSRA signed on to during September:

• Letter urging Congress for a five-year extension of Hospital at Home (9/3)
• Letter in support of the Give Kids a Chance Act of 2025 (H.R. 1262/ S.932) (9/10)
• Letter in Support of FAIR Model and JAG Legislative Asks (9/22)

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.

This month, we honor and remember:

Leonidas (Leo) James Ballard-Van Gundy

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.

BDSRA earns about 20% on See’s Candy items purchased. 

Fundraiser name: BDSRA Sweet Support for Batten

Time window: Starts September 25, 2025, and ends December 5, 2025

Click here to see the candy that’s for sale! 

Click here for the Shop link. 

Are you planning a birthday fundraiser for BDSRA this year? Now is the time to start planning, and we are here to help!

Facebook is no longer paying the credit card fees for nonprofits and has turned over the program to another entity to manage. BDSRA is not currently eligible for their program, but we are working on it.  

The good news is that BDSRA can make a custom link and QR code for you to create a birthday fundraiser that directly comes to us! Creating a Fam Fund is another great way to raise funds in celebration of 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 contact Heather Dainiak at heather@BDSRAfoundation.org to get started.

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.