Dear Batten Community,

In 2023 we awarded $71,800 for families through our grant programs, including the Fore The Journey funding provided by the ForeBatten Foundation. This year, to date, we have awarded more than $24,600. Unfortunately, we are entering the 4th quarter with zero funds available for these programs. As a result, we must pause processing new grant applications.

We are grateful for every donation. However, this shortfall is the result of too few donations to our greatest needs funds. This type of donation empowers us to meet the greatest needs in our community, and the grants are a primary vehicle to do this.

Shortly we will launch our annual giving campaign, but you don’t have to wait to receive materials in the mail. We urge you to go to our website and donate to the greatest needs fund today. This will help us fund these very important grants for our families in need.

Top two photos: BDSRA Database Manager Noah speaks on the Rare Sibling Panel at the Global Genes RARE Advocacy Summit on Friday, September 27, 2024.

Bottom left: Noah (left) is pictured with his mother, Jennifer (right), who serves as the Director of Community Engagement for the Courageous Parents Network.

Bottom right: Noah (right) is pictured with Effie Parks (left), the creator and host of the Once Upon A Gene podcast. Parks is a rare disease mother to her son Ford, who has CTNNB1 syndrome.

KANSAS CITY, Mo. – The BDSRA Foundation was represented at this year’s Global Genes RARE Advocacy Summit in Kansas City, Missouri. BDSRA Database Manager Noah Siedman attended the event and was featured as a speaker on the Rare Siblings panel.

Click to read the full report below as Siedman analyzes the sessions he attended, his message to the audience during the Rare Siblings panel, and his overall takeaways from the event and how they can help the Batten disease community.

We applaud the news from our friends at ForeBatten Foundation that the FDA has provided IND clearance for the clinical investigation for Zebronkysen (FBF-001) under the N-of-1 guidance for individualized Antisense Oligonucleotide (ASO) drug products. Two patients with the unique CLN3 c.569dupG mutation began treatment in June 2024.

To read more about this significant milestone, please click the button below.

To learn more about ASOs, check out this 2-minute clip from Harvard Medical School.

The BDSRA Virtual Memorial is undergoing maintenance so that it can continue to be an enduring place of remembrance and celebration of our Batten community. We apologize for the downtime and want to assure the community that all memories previously stored are secure during this process.

We appreciate your patience and trust in us to steward this project. If you have any questions, would like to create a memorial page, or make updates to an existing one, BDSRA staff is happy to assist. Please contact Noah Siedman at noah@bdsrafoundation.org.

The Cardiac Neurodevelopmental Program at Boston Children’s Hospital announced the next Cardiac Neurodevelopmental Family Symposium, which will feature BDSRA Database Manager Noah Siedman!

This event brings together families and professionals from the heart defect community for a day of learning, networking, and building connections.

When: November 1-2, 2024

Where: Boston Children’s Hospital, main building and Enders building

Cost: $35 (BCH employees should email us directly for complimentary admission)

How to sign up: (Click the button below)

Tickets are limited.

September was Newborn Screening Awareness Month. Newborn screening (NBS) is widely recognized as one of the largest and most successful disease prevention programs in U.S. history. BDSRA continues to advocate for NBS and Recommended Uniform Screening Panel (RUSP) alignment legislation to allow early intervention and help save lives.

Watch the Toolbox Tuesday video below and click the button to learn more.

The current relationship between Batten disease, the Recommended Uniform Screening Panel (RUSP), and newborn screening can be difficult to understand. The video above breaks down these concepts and what they mean to the Batten community.

Visit the links below, courtesy of the EveryLife Foundation for Rare Diseases, for more Newborn Screening tools and resources.

• Rare Disease Congressional Caucus briefing on “Innovating and Strengthening the Newborn Screening Program”
• Newborn Screening Bootcamp 2024 sessions
• RDLA Monthly Webinar: Discussion on Newborn Screening and Diagnostics for Rare Diseases
• Visit our action center to learn more

Your gifts to BDSRA make a real difference for our Batten families. Thanks to generous donors, 49 people from 16 different Batten families could attend this year’s Annual Family Conference in St. Louis this past July.

From the bottom of our hearts, thank you! 💚

Select presentations from the 2024 Annual Family Conference in St. Louis are publicly available on our YouTube channel! Recordings include the Science & Research session, Celebration of Life, Dinner Dance announcements, financial sessions, caregiver support presentations, and much more!

SSA Talks is an audio series where you can hear from Social Security experts about important Social Security topics.

This episode features Kathleen Romig, Senior Advisor in the Office of the Commissioner, sharing the types of benefits Social Security offers to children, which include survivor benefits, Supplemental Security Income, child benefits, and Social Security Disability Insurance Benefits for Youth with Disabilities.

Click the button below to listen!

“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.

• Letters to Congressional leaders supporting the Creating Hope Reauthorization Act and reauthorization of the Rare Pediatric Disease Priority Review Voucher program at the FDA:
  • House letter
  • Senate letter

On July 17, FDA announced the establishment of the FDA Rare Disease Innovation Hub.

The Reagan-Udall Foundation for the FDA, in collaboration with FDA’s Rare Disease Innovation Hub, will host a public meeting on Wednesday, October 16. The meeting will bring together rare disease patient advocates, academic researchers, regulated industry, and other key stakeholders to discuss how the recently announced Rare Disease Innovation Hub can best engage with members of the rare disease community and prioritize its work.

In-person participation will be at the FDA’s White Oak campus, a live stream will be available, and written comments will be accepted until October 31. Prior registration is required and limited.

• New California Law Establishes the Jacqueline Marie Zbur Rare Disease Advisory Council (RDAC)

Grab your costumes and join us for a frightfully FUN time at the 2024 Boo Bash taking place on Sunday, October 13, at 4 PM EDT, via Zoom! This virtual party allows parents and SIBs to gather with their loved one(s) with Batten disease and enjoy a Halloween party with fellow Batten families and the BDSRA staff.

FREE party kits are available for families to order during registration until 11:59 PM EDT on Sunday, October 6, while supplies last. The party kit will include a story to read and craft to create during the party. Party kits are reserved for U.S. and Canadian families only. You may still register to attend the party after the kit deadline passes.

As part of our Life Goes On program for bereaved Batten parents, the next BDSRA virtual Grief Chat will be held on Sunday, October 20 at 7 p.m. EDT. If you’ve already completed the Grief Chat form, you will receive an email reminder one week before and the day of 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.

Sayra Aykaç | June 21, 2011 — September 17, 2024

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.

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