We’re over halfway there! We can’t thank you, our Batten community, enough for your advocacy. As June 9 nears, learn how you can advocate for International Batten Disease Awareness Day by checking out the activities (some of which begin TOMORROW, June 1).

Batten facts, race bibs, letters to your reps, sharing your story, and raising funds. There are numerous ways to get involved leading up to International Batten Disease Awareness Day on June 9! Click the button below to learn more.

Friday, June 14 is the deadline for the Early Bird Registration Special, conference T-shirts, registration for offsite events, and submissions for the Celebration of Life.

Take advantage of the Early Bird special that includes $5 off the $25 price of each adult registration by June 14.

Register, view the agenda, activities for SIBs and Life Goes On, hotel and travel information, and more by clicking the button below. Be sure to follow weekly posts on our social media channels showcasing activities and presentations planned for this year’s conference!

Click the button below to register for the conference and scroll below to the “Upcoming Events” section for information on pre-conference virtual events for moderator support training, first-time conference attendees, and SIBs.

Left: A presentation is given at the Sanford/PPALS Pact. Right: Sharon King, a bereaved Batten mother, advocate, and President of Taylor’s Tale presents at the EveryLife Foundation Scientific Workshop. 

In her own words, BDSRA Foundation President & CEO Amy Fenton Parker answers questions and breaks down her takeaways from the Sanford/PPALS Pact and the EveryLife Foundation Scientific Workshop. 

PPALS

What event did you attend and where did it take place? 

Held at Sanford Research Center in Sioux Falls, South Dakota, PPALS is a two-part certification program. Its acronym stands for Professional Patient Advocates in Life Sciences, and our Board Vice Chair Barbara Wuebbels founded it, along with a few of her colleagues in the field. I was honored to receive a travel stipend and scholarship so that I could attend!

I first attended in 2022 and returned to finish my certification this May. The program brings together industry partners who work in patient advocacy and patient advocacy organizations to learn about how to better serve our communities, learn about each other’s roles and responsibilities, and create a space for robust conversation on how to work better together.

What new information did you learn, and how can that information help benefit/educate the Batten community?

I learned more about industry partnerships—especially what they are and are not allowed to share with patient organizations. I better understand the role they play within their organization and that for many, they are truly advocating for the communities they serve within their organization. This helps me to understand their budgeting processes better, and how they can help BDSRA and our community.

I learned a lot about epidemiology and investment in rare disease. These two presentations sparked great conversations! I can see the benefit of investing in our own epidemiology study so that we can truly understand where and how many families we have with Batten disease of all CLN types. This would further our opportunity to reach out to industry partners and researchers and encourage study and investment in Batten disease. I am already following up on this.

Who did you have conversations with? What was the primary theme of your conversations with fellow attendees?

I learned much from the other Patient Advocacy Organizations and Industry Patient Advocates. The program split us into separate groups but brought us together for case studies to discuss working together. These were fruitful conversations. By listening to industry advocates, we learned about their perspectives on working with organizations and families. We also shared the family and organization’s insight into the case studies to demonstrate our position with them.

The conversations with my peers were equally productive. We discussed various topics including marketing strategies and fundraising ideas. We talked about board development and governance, including an annual checkup. We also discussed public policy and legislative action, so that we can build stronger relationships with representatives and agencies. Our group was small, so we had plenty of time to talk and attention from the facilitators.

What were some of your other takeaways from this event?

The biggest takeaways for me were epidemiology and understanding how industry has changed its strategy over the years to follow profitable drug development. Cell and gene therapies have not brought in the income that was once projected to earn. Research and development look very different than they did just 20 years ago. We must change our approach to producing treatments and cures for Batten disease and other rare diseases.

Continue reading by scrolling below!

Whether it’s an urgent need, joy along your Batten disease journey with the help of Fore the Journey, or sending equipment to another family in need, BDSRA is here to support you through our family grants.

The EveryLife Foundation for Rare Diseases offers financial resources and medical resources for the rare disease community.

Learn more by clicking here.

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.

Left: Fenton Parker and BDSRA Board Vice Chair Barbara Wuebbels pose for a photo at the Sanford/PPALS Pact. Right: BDSRA Head of Research & Medical Affairs Dr. Ineka Whiteman tunes into EveryLife Foundation Scientific Workshop during night-time in Australia.

On Wednesday, June 5 at 8 p.m. EDT, Dr. Joseph Vigil will lead virtual training via Zoom for anyone who wants to facilitate discussions in the breakout groups at the Annual Family Conference in July. The breakout groups at the conference will include bereaved fathers, bereaved mothers, grandparents, and actively caring mothers and fathers by CLN type.

Join us as we celebrate Batten families around the world, advocate, and raise awareness and funds! BDSRA is hosting the 2024 International Batten Disease Awareness Day Virtual 5K on Sunday, June 9th, 2024.

You don’t have to participate in the virtual 5K to advocate for Batten disease. This year, we’re providing a variety of activities for you to raise awareness and funds for Batten disease and BDSRA.

Learn more about the activities, how to donate, and how to get involved by clicking the button below.

BDSRA will hold a First-Time Conference Attendee Orientation on Tuesday, June 18, at 6:30 p.m. EDT. This virtual event will be a casual forum for those attending the Annual Family Conference for the first time this July. Attendees will receive more information about the conference during this event and can ask questions.

Before you reunite with your fellow SIBs in person at July’s Annual Family Conference in St. Louis, catch up with them and meet new SIBs! Whether you’re new to the SIBs program or a longtime member, we encourage you to attend the SIBs Warm-Up on Wednesday, June 19, at 6:30 p.m. EDT.

The next BDSRA Grief Chat is scheduled for Sunday, June 23, at 7 p.m. EDT.

Prior registration is required for newcomers. If you’ve already completed the Grief Chat form, you will receive an email reminder one week before and the day of. Click the button below for more information.

Where will the path with Batten disease take you? We hope to St. Louis this summer!

Meet us in St. Louis for our Annual Family Conference, which will feature sessions on research, advocacy, caregiver support, grief, and activities for SIBs, bereaved parents, and more! Plus, meet new members of the Batten community and reunite with families you’ve met along your journey.

Click the button below for more information.

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: 

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.

On May 23, the BDSRA Foundation hosted a virtual roundtable meeting with 19 key stakeholders across four continents taking part in a robust discussion on the future direction of CLN1 natural history studies.

It was encouraging to see such active participation and equal voices around the table, with representatives from the CLN1 family community (U.S., Europe, and Scandinavia), patient advocacy groups (BDSRA Canada, BDSRA Australia, BDFA UK, and Taylor’s Tale), industry partners (JCR Pharmaceuticals, Taysha Gene Therapies, and Collaboration Pharmaceuticals), and clinical centers involved in CLN1 natural history studies, including University Medical Center Hamburg-Eppendorf, Germany; Rush University Medical Center, Chicago, IL; Kennedy Krieger Institute, Baltimore, MD; Nationwide Children’s Hospital, Columbus, OH; and University of Rochester Batten Center (URBC), Rochester, NY.

Industry partners briefly shared updates on their pipeline programs, including those in clinical and preclinical stages. This laid the foundation for the following discussions, reinforcing the need for and importance of robust natural history data in driving CLN1 therapeutic development. Existing CLN1 natural history data collected through respective studies in the U.S. and Germany were then presented to the group. Discussions that followed focused on critical learnings and insights gained from these studies, brainstorming how to best complement and/or fortify those current datasets across continents, countries, and sites, and how to engage and collaborate with CLN1 patients and families worldwide effectively. Participants agreed that natural history study activities moving forward must be consistent and unified across continents and sites to ensure the best outcomes for our patient community. The next big step will be enacting the key ideas and potential solutions discussed. There is more to come, and we look forward to sharing further developments soon!

Help Shape The Future of Batten Disease Research – Community Survey Phase 2 Opening Soon

Over the past two weeks, you may have seen announcements on social media and in your inboxes seeking your input on the topics and questions YOU feel should be prioritized by Batten disease researchers and research funders.

We are delighted to partner with Kennedy Krieger Institute on this two-phase Research Priority Setting project and want to thank all those who participated in the Phase 1 survey.

We’re halfway there!

In Phase 2, we will collate all the responses received in Phase 1 and produce a summarized list of research questions. This list will be sent around in a second survey, and we will ask stakeholders to rank the list of research questions in order of importance to YOU. Those rankings will determine the “top priority research” questions that will help inform and drive research priorities and future investment in Batten disease. You may participate in Survey 2 whether you completed Survey 1 or not. Whether you’re a parent, clinician, researcher, industry representative, policy maker, educator, allied health, or support worker, we are seeking input from all stakeholders.

Your role in the Batten disease community is important, and your response matters.

So please keep an eye out for the Phase 2 survey coming to your inboxes and social media channels soon! And as always, thank you for your support and dedication to our shared mission.

Warm regards,

Click the button below to read Dr. Whiteman’s full column, which includes information on where REGENXBIO’s CLN2 gene therapy program preliminary data was recently presented, a research opportunity, publication summaries, and more.

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

Brixton William Tice | April 10, 2018 — May 1, 2024

Ahsen Köker | December 19, 2012 — May 4, 2024

Eymen Dalkıc | January 23, 2018 — May 4, 2024

Maddison Nicole Ferran | June 26, 2015 — May 7, 2024

Reece Duncan Mitchell | July 6, 2013 — May 10, 2024

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

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

NOTE: This donor list doesn’t include donations made to the International Batten Disease Awareness Day fundraiser. Those donors will be recognized in the June newsletter.  

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.

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