It’s almost that time! It’s not too late to register and attend the Annual Family Conference in Lincoln, Nebraska, from July 11-13! Registration does not close; however, the deadline to register for events and meals has passed.

We hope to see you soon for a weekend filled with research updates, research studies, music therapy, pet therapy, breakout groups, a FREE dinner dance, and more! Scroll down for more important information, and click the button below for more conference information.

A big THANK YOU to all of our donors, Fam Funds, families who hosted fundraising events, and everyone who shared our posts and advocated through our International Batten Disease Awareness Day activities! Your continued gifts, support, and advocacy are crucial so that BDSRA can continue to provide support to families in need, source research for treatments and cures, and advocate for families’ healthcare access and equity. With your continued support, we will accomplish this.

The Vanguard Clinical Rare Disease Foundation (VCRDF) has officially launched development of VCRDF-CLN3, a precision antisense therapy targeting the most common CLN3 variant.

Endorsed by the ForeBatten Foundation and developed in collaboration with Dr. Michelle Hastings (University of Michigan) and Dr. Yael Shiloh-Malawsky (Batten Disease Center of Excellence Affiliate, UNC), this effort builds on the groundbreaking Zebronkysen program and has already received early alignment from the FDA.

Read the press release below.

We look forward to hearing from Tiffany Sepp and Dr Shiloh-Malawksy at the Annual Family Conference in Lincoln, Nebraska, next Friday. Congratulations to the entire research team, Tiffany Sepp, and the Vanguard Clinical Rare Disease Foundation!

Tern Therapeutics announced that TTX-381 received Regenerative Medicine Advanced Therapy (RMAT) Designation from the FDA. Read the full press release and patient community letter from Tern below.

Last month, Theranexus and the Beyond Batten Disease Foundation (BBDF) announced new real-world data strongly supporting the efficacy of Batten-1 (miglustat) as a treatment for CLN3 disease. Click below to read the full press release.

The article ‘Neuronal Ceroid Lipofuscinoses Overview’, published in GeneReviews last week.

Co-authored by our own Dr. Ineka Whiteman and colleagues in the U.S. Batten Disease Clinical Centers of Excellence, the article provides an overview of clinical characteristics and genetic causes of all Batten disease (NCL) subtypes. It also discusses clinical evaluation strategies to identify the genetic cause of NCLs, review of management, and approaches to informed counseling of family members of affected individuals.

Click below to read the publication and see the full list of authors.

Study Title: Characterizing Sleep in Batten Disease
Principal Investigator: Heather Adams, PhD
Research Sponsor: BDSRA Foundation

The University of Rochester Batten Center is conducting a study of sleep function in individuals with CLN2 and CLN3 Batten disease.

What is involved?
Affected individuals will provide saliva samples to test melatonin concentration and wear an actigraph (i.e. a wrist-watch style activity monitor). Parents/caregivers will assist the affected individual with study activities and will complete questionnaires.

Who may be eligible? Affected individuals who…
• have a confirmed genetic or enzyme-based diagnosis of CLN2 or CLN3 disease
• have any symptoms of CLN2 or CLN3 disease
• are at least 2 years old
• live at home with at least one primary caregiver
• have not taken oral melatonin (supplement), or are able/willing to stop taking melatonin during study participation (approx. 9-12 days).

No travel is required! All study activities will take place at your own home.

The affected child & parent will receive $200 (total for household) for participation in the research.

If you are interested in learning more about the study, please contact the study team at BattenSleepStudy@URMC.Rochester.edu or call study coordinator Marianna Pereira-Freitas at (585) 274-0205.

On behalf of the NCL2025 Organizing Committee, BDSRA Australia is thrilled to be hosting the 19th International Congress on Neuronal Ceroid Lipofuscinosis (NCL) in the stunning surrounds of the Sunshine Coast, Queensland, Australia. As the premier conference on Batten disease, the bi-annual NCL Congress brings together world-leading researchers, healthcare providers, industry partners, innovators, and patient advocacy leaders from around the globe.

John Ireland is currently the SIBs representative on the BDSRA Foundation’s Board of Directors and has been working with the SIBs program for over 30 years.

Watch the video above to learn more about him!

According to the EveryLife Foundation for Rare Diseases, the newest Congressional Budget Office (CBO) estimates a historic Medicaid hit, which could cause more than 11.8 million Americans to lose coverage — especially our rare disease and disability communities.

The Senate version of the Reconciliation bill slashes $930 billion from Medicaid — an increase in cuts over previous versions of the bill. The bill passed the Senate by a 51-50 vote on Tuesday, July 1. The bill has to pass the House again before President Trump can sign it into law.

Whether you, your loved one, or someone you care for receives Medicaid, we must ALL Act NOW. Medicaid is a vital resource for our rare disease community. This will be a catastrophic resource loss for our community.

Contact your House reps and tell them NO. Click below for instructions.

The BDSRA Foundation stands with international partner BDSRA Canada, Batten parents, Jori Fales and Trevor Pollock, and their daughter, Charleigh, who has CLN2.

B.C. Health Minister Josie Osborne announced on June 18 that the family’s coverage for Charleigh’s Brineura treatments would be discontinued, based on a recommendation from Canada’s Drug Agency.

Our Head of Research & Medical Affairs, Dr. Ineka Whiteman, has spoken to numerous local media outlets in Canada to educate others and advocate for Charleigh.

Click the button below to learn more.

Click below to read the letters BDSRA signed on to during May and June:

• Letter to appropriation committees expressing concern over the President’s Fiscal Year 2026 (FY26) budget request that proposes cutting the NIH budget by $18 billion dollars and summarily consolidating or eliminating institutes and centers without congressional hearings or public input
• Letter to Commissioner Makary of the FDA, calling for an interactive town hall-style meeting series to strengthen engagement between the FDA and patient advocacy groups
• Letter recommending at least $51.303 billion for the National Institutes of Health (NIH) for fiscal year (FY) 2026, in addition to support for the Advanced Research Projects Agency for Health
• Letter advocating for the preservation of the Ohio Rare Disease Advisory Council

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

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.

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.

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 Beth Fries 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. 

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