July 2024 Volume 35, Issue 7
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2024 Annual Family Conference Recap
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The 2024 Annual Family Conference is in the books and it was one to remember! We’d like to thank everyone who joined us in St. Louis. No matter who you are or where you’re at in your Batten journey, we’re grateful for this July weekend each year that brings the Batten community together.
We hope you join us again next year in LINCOLN, NEBRASKA!
LOTS of news was unveiled at the conference’s Saturday night Dinner Dance! Scroll below to the “Latest News” section to learn more.
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View the Full Conference Photo Gallery!
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Participate in the NCL Researchers Challenge by THIS SUNDAY!
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Batten disease researchers worldwide were invited to enter a three-minute thesis-style video presentation, summarizing their team’s main research approach, the latest findings, and the significance of these findings for our Batten community as part of this year’s NCL Researchers Challenge.
There are nearly 20 videos that you can watch on the conference app! Conference registrants are invited to view and vote for their favorites.
The deadline to vote is Sunday, August 4.
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Watch Select Conference Presentations
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Select presentations from the 2024 Annual Family Conference in St. Louis are available in the conference app! Recordings include research & support sessions, Saturday’s Dinner Dance presentations, and the Celebration of Life.
To view the recordings in the conference app, click on the agenda icon, and you’ll see “View Recording” under select presentations. To watch the Saturday Parade and Dinner Dance presentations, click on “Dinner & Dancing” and click the links in the session description.
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Thank You To Our Conference Sponsors!
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BDSRA Hires Heather Dainiak as Director of Family Support
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The BDSRA Foundation and BDSRA Canada are excited to bring Heather Dainiak on as our new Director of Family Support beginning August 1. Heather has lived experience as a Batten Advocate! She is wife to Chris and mother to William, an active SIB, and Nicholas, who lived with CLN2 before Brineura was available. She supported Nicholas through a clinical trial and co-founded their family foundation. She helps organize a translational NCL scientific conference every two years and has served for more than 10 years as a key leader of the childcare provided at the BDSRA Annual Family Conference. Heather’s experience will serve our U.S. and Canadian families well.
Join us in welcoming Heather to the team!
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BDSRA Accepting New Board Member Applications
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The BDSRA Foundation Board of Directors is accepting applications for new members until August 31, following the Board By-Laws. Advocates who are experienced in Batten disease, bi-lingual, and have experience in education, law, fundraising, or advocacy are encouraged to apply.
All applications will be reviewed and some may have phone interviews. Elections will be held at a fall board meeting, and all applicants considered will be notified of the decisions made.
Click the button below for more information.
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FDA Approves BioMarin’s Brineura for Children Under 3 Years with CLN2 Batten Disease
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On Wednesday, July 24, BioMarin announced that the FDA approved Brineura for children under 3 years old with CLN2 Batten disease. Click the button below to read the full press release from BioMarin and scroll down to read more details in Dr. Ineka Whiteman’s monthly research column.
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BDSRA Announces Centers of Excellence & Affiliates and Batten Disease Global Research Initiative
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BDSRA announced its Center of Excellence & Affiliate members and the formation of the Batten Disease Global Research Initiative during the Saturday night Dinner Dance at the Annual Family Conference in St. Louis. Click the buttons below and scroll below to read Dr. Ineka Whiteman’s column for more details.
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Together, the BDSRA Foundation and BDSRA Canada, with our Global Research Initiative (GRI) partners, is delighted to announce the inaugural GRI Grant Round is now inviting Expressions of Interest.
We seek to support the most promising research ideas worldwide that address key research questions and areas of unmet need in Batten disease. Submissions CLOSE AUGUST 30. To find out more about the Program and how to apply, please click the button below.
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Apply for BDSRA Support Grants Today
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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.
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The EveryLife Foundation for Rare Diseases offers financial resources and medical resources for the rare disease community.
Learn more by clicking here.
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Complete The Family Register Form
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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.
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BDSRA Signs On To 3 Letters
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Click the links below to read the letters BDSRA signed on to in July:
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Join FDA CBER’s Upcoming Patient Listening Meeting – September 20, 2024
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| The FDA’s Center for Biologics Evaluation and Research (CBER) will host a patient and care partner listening meeting – Patient and Care Partner Perspectives on Safety Considerations for Approved Gene Therapy Treatments for Rare Diseases on Friday, September 20 from 11:00 a.m.–4:30 p.m. EDT.
The objectives of this listening meeting are to hear from patients and care partners on their perspectives on short-term and long-term risks of approved gene therapy products, to learn what types of information patients would find helpful in their decision-making when considering gene therapy, to learn about their considerations and experience of participating in post-market long-term follow-up studies, and to help inform patient-centered protocols for long-term follow up of gene therapy products.
The meeting will be divided into two sessions and patients, advocates, and caregivers are encouraged to request to speak during the registration process. |
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FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients
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The FDA plans to establish a Rare Disease Innovation Hub (the Hub). The Hub will work across rare diseases but will especially focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood.
Click the button below to read the full press release from the FDA.
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Dog Days of Summer Virtual Party
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Sunday, August 11 | 4 PM EDT
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Gather your pups and pals because it’s almost time for the 2nd Annual BDSRA Dog Days of Summer Party!
This virtual party will take place via Zoom on Sunday, August 11, at 4 PM EDT. The deadline to reserve party kits has passed, but you may still register to attend and join the fun. A Zoom link will be sent to the email you registered with.
Click the button below to register!
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Sunday, August 18 | 7 PM EDT
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As part of our Life Goes On program for bereaved Batten parents, the next BDSRA virtual Grief Chat will be held on Sunday, August 18 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.
If you haven’t attended a Grief Chat this year, please click the button below to register.
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Do You Follow BDSRA on Social Media?
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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:
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Research Updates with Dr. Ineka Whiteman – July Edition
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Annual Family Conference 2024 – Science and Clinical Program Highlights
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What a weekend!! I was absolutely blown away by the atmosphere, sense of community, robust discussions, and engagement of attendees this year. This is my third consecutive year attending the conference in person and I can honestly say, it feels bigger and better every year.
The conference kicked off with the main Research Session on Friday morning. Presentations covered Batten research advances “from lab bench to bedside,” including cell and animal models, biomarker discovery, and preclinical therapies. The session finished with the latest clinical trial updates and an open, lively Q&A session with academic and clinical researchers, and biotech company representatives. Day two kicked off with the popular 90-minute “Ask-A-Doc” session, where families could ask impromptu questions to a panel of six Centers of Excellence (CoE) and Affiliate Center directors. In the afternoon, more than 20 clinicians representing nine Centers came together for its second in-person Center of Excellence Working Party meeting to continue developing collaborative opportunities and network projects to advance clinical education, research, and best practices in Batten disease care.
Other science and clinical program highlights throughout the weekend included concurrent sessions on “Behavior, Cognition and Learning,” “Palliative Care: Debunking the Stigmas,” Case Management 101 and the “Ask Me Anything: Scientists Edition” with research scientists who were delighted to share a collection of laboratory gadgets and research tools for attendees to try out, and to answer a variety of curious questions on their laboratory research. Our sincerest thanks and appreciation to all speakers and panelists who willingly offered to contribute their time and expertise to this robust program. And my gratitude to Dr. Jon Cooper and Dr. David Pearce for their help in pulling together some of our best science, clinical, and SIBs sessions yet!
Some sessions mentioned here were recorded and are now available in the conference app.
A huge, heartfelt thanks to all our families who participated in the many onsite research opportunities during the conference. Our researchers are so grateful for the invaluable opportunity to have met and worked with so many families in person. And of course, an enormous shout out to the research teams for your time and dedication to the advancement of Batten research, and in particular, Dr. Jill Weimer and her team from Sanford Research who coordinated the mountain of logistics to make this happen.
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Remember those we have lost to Batten disease. We work daily to build a brighter future for families in their honor and memory.
Megan Elizabeth Dilley | July 7, 2010 — June 16, 2024
Kerim Şeviş | July 5, 2013 — July 5, 2024
Ryker Allen Pfingsten | June 1, 2012 — July 15, 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.
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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.
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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.
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To view the list of recent donors, please click the button below!
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Give to the $5 Fridays Program!
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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.
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That’s the power of FIVE.
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Sign Up For The Fam Funds Program
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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
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We connect your donation to Support, Research, and Advocacy for families of all CLN types.
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