BDSRA Foundation

BIG RESEARCH NEWS! Following a robust selection process, the Batten Disease Global Research Initiative has awarded research grants to six recipients totaling USD $320,000! Tune in next week as we unveil our grant recipients: https://battenresearch.org #BattenResearch #BrainAwarenessWeek #BattenDisease #RareDisease #Science #Research #Clinical #Grant

? Join our Virtual Coffee Chats this month! ? Hosted by Batten parent Heather Dainiak, these chats are a safe space for parents and caregivers of all CLN types in the United States, Canada, and abroad. This month’s theme is, “The Power of Memories.” Join us on Tuesday, March 25 at 7 PM EDT or Friday, March 28 at 11 AM EDT. If you registered for last month’s chats, you do not need to register again and will receive email reminders: https://ow.ly/8afg50Vguz9 #BattenAdvocatesForACure #BattenDisease #Coffee #Chat #Support #Virtual

IT’S BACK!! ?? For the third straight year, we are holding another T-shirt contest to determine the design of this year’s International Batten Disease Awareness Day T-shirt! DEADLINE IS 11:59 PM EDT ON SUNDAY, MARCH 30. View the complete rules and guidelines: https://ow.ly/fR3l50Ve7mW #BattenDay2025 #BattenAdvocatesForACure #BattenDisease #Awareness #Advocacy #Contest

It’s Brain Awareness Week – a global campaign to foster public enthusiasm and support for brain science. Let’s take some time to learn more about the Batten Disease Global Research Initiative. Big news is coming soon! ?? Visit the GRI website: https://ow.ly/ew3n50VeS6J #BattenResearch #BrainAwarenessWeek #BattenDisease #RareDisease #Science #Research #Clinical

?? RARE DISEASE WEEK RECAP ?? Batten grandfather Mitch Larson traveled to D.C. from Nebraska last week to participate in Rare Disease Week on Capitol Hill. Listen as he details his experience advocating for the Batten disease community. Visit our website for more content from Rare Disease Week, and THANK YOU ALL for your relentless advocacy: https://ow.ly/wn0O50VcCq8 #BattenAdvocatesForACure #RareDC2025 #BattenDisease #RareDisease #Advocate

It's late in the day, but today has been World Rare Disease Day. I wasn't able to repeat the awareness campaign that I undertook last year. However, I didn't want the day to pass without sharing something that is hugely important. Many of you know that Batten disease is very close to my heart. During my awareness campaign last year, I featured an article about Ollie and Amelia. They are brother and sister who both have CLN2, the late-infantile form of Batten disease. While there is no cure, there is a very effective enzyme replacement treatment that slows down disease progression. It is a lifeline for families trying to give their children the best quality of life possible for as long as possible. However, last year, Amelia and Ollie's mum, Lucy, explained that in 2018/19, the National Institute for Health and Care Excellence (NICE) stated that although the treatment was working, due to the lack of long-term evidence, they deemed the drug too expensive.? ? They launched a judicial review against this decision, which resulted in NICE agreeing to a Managed Access Agreement for a period of five years. This allowed the drug company to collect long-term data and continued access to the drug for those who met the criteria. This agreement ended in November 2024. However, there still remains indecision on whether this treatment will become available on the NHS, meaning that families in the UK are in limbo. Lucy says that, "We need as many people as possible to join us at the Houses of Parliament…we have to make a stand, we need to be seen, we need to be heard". The CLN2 Community and @bdfabattendisease will be gathering outside the Houses of Parliament on Wednesday, 12th March (1-3 pm) to raise awareness of our campaign to ensure that Brineura is approved by NICE. Please come along and join us as we fight for the right for every child with CLN2 to receive this life-saving treatment now and in the future. Exact location to be announced soon. Read more on Amelia and Ollie's story here. https://lnkd.in/eda2835v Or follow them on Instagram (@ameliasarmy) This story is also being played out in British Columbia, Canada, where another young child with CLN2 is under threat of treatment being withdrawn largely based on cost. We must do more to secure these treatments to ensure the quality of life, and extended life for children with rare diseases. #Battendisease #raredisease #awareness #sanfilippo #rarediseaseday Rare Disease Day Batten Disease Support & Research Association (BDSRA) Australia BDSRA Foundation

From the bottom of our hearts, THANK YOU! ???? To everyone who ordered Panda Express, shared our posts to spread the word, and told family, friends, co-workers, neighbors, etc., your advocacy makes a difference to the Batten disease community—a big thank you to the Palermo family for setting this fundraiser up. Thank you, everyone, for raising funds, supporting Batten families, and advocating for treatments and a cure. Panda Restaurant Group #BattenAdvocatesForACure #PandaExpress #BattenDisease #Donate #Fundraise #RareDiseaseDay #RareDC2025

?? We are now up to 19 Fam Funds! ?? Welcome our newest Fam Funds and check out their pages! ?? This initiative allows Batten families to share their stories and raise funds within their communities for a mission area of their choice. Learn more and get started: https://ow.ly/z0Ug50V834X #BattenAdvocatesForACure #FamFunds #BattenDisease #Fundraising #RareDisease #Donations

The February edition of The Illuminator Newsletter will not be published this week. News and notes that would’ve appeared in that edition will be included in a combined February/March edition. Sign up for our email list to receive our monthly newsletter and important updates: https://ow.ly/qxgR50V82Ql #BattenAdvocatesForACure #BattenDisease #RareDisease #TheIlluminator #Newsletter #News #Notes

ADVOCACY IN ACTION: Our Database Manager Noah Siedman traveled to Bethesda, Maryland today and spoke about his experience as a Rare Disease sibling at the Rare and Ready Meeting. YOU can advocate on this Rare Disease Day by using our advocacy module: https://ow.ly/cPHs50V8Jjw #BattenAdvocatesForACure #RareDC2025 #BattenDisease #RareDisease #RareDiseaseDay #Advocate