Advocacy Toolkit
Each year BDSRA staff, board members, and parent advocates travel to Washington, D.C. to meet with legislators and appear before committees to push for accelerated drug discovery, increased services for those with rare disease, and improved FDA processes and other initiatives important to our families. Along with the Rare Disease Legislative Advocates, we join with other patient advocacy groups to speak for those who cannot.
BDSRA board, staff, and volunteers have also advocate across state legislatures during Rare Disease Day and Batten Awareness Weekend to raise awareness and increase research related funding for NCL studies. Parents of children with Batten disease have testified to committees in numerous state legislatures on a range of issues, including access to medications, accessibility, and support for services to those caring for patients with life limiting illness.
In 2013, Batten disease advocates appeared on program panels of the National Institutes of Health and National Institute of Medicine to discuss medical and ethical ramifications of gene therapy in rare disease research. In February 2014, our Batten team from BDSRA, Taylor’s Tale and Noah’s Hope visited over 40 Congressional offices on Rare Disease Day to talk about the care needs of Batten children and push for drug discovery.
Parents and board members also have testified before the FDA during public meetings and panels on patient and caregiving challenges for rare disease. Legislative alerts, testimony and panel opportunities, and FDA updates are made available to the Batten community through BDSRA communication channels.
BDSRA actively engages in educational initiatives within the medical community nationally to inform and educate physicians, neurologists, nurses, allied health professionals, genetic counselors and palliative care specialists about Batten disease, the diagnostic challenges, and research and support services available to families.
Resources for rare disease advocacy also include:
Rare Disease Week
Click here to learn more information about Rare Disease Week on Capitol Hill, occurring February 28th-March 2nd, 2023.
State/Local Initiatives
Click here to learn more about finding your State Representative, using your voice, and how BDSRA can help.
Hometown Efforts
Click the icon below to learn more about how to fundraise, resources for contacts, and how to share your story.
June 9th International
Batten Awareness Day
June 9th of every year is designated as International Batten Awareness Day. Families, their advocates, and supporters bring the message of hope and awareness of the disease to thousands of people and inspire charitable gifts to BDSRA. Through social media campaigns, hometown fundraisers, school and church events, and awareness projects, volunteers explain their connection to Batten disease and the importance of raising funds for support and research. BDSRA hosts an annual fundraiser that raises funds to help further our mission.
2024 International Batten Disease Awareness Day
Not only did we reach our $75,000 fundraising goal in 2024, we surpassed it by over $9,000! We here at BDSRA continue to be amazed by our Batten community’s determination, persistence, and support. Thanks to you, we can provide five travel stipends for our community to attend Rare Disease Week on Capitol Hill, continue to support Batten families through grants such as the Equipment Exchange Grant, engage the community through virtual events, and much more. From the bottom of our hearts, THANK YOU!
Recent News
FDA Approves BioMarin’s BRINEURA® for Children Under 3 Years with CLN2 Batten Disease
Read the following press release and information below courtesy of BioMarin. Read the original press release by clicking here. SAN RAFAEL, Calif., July 24, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. [...]
NEW Upcoming Batten Disease Community Event; 2025 Family Conference Location
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 [...]
BREAKING: BDSRA Foundation Unveils CoE & Affiliates; Batten Disease Global Research Initiative
ST. LOUIS (July 15, 2024) – The BDSRA Foundation announced its Clinical Center of Excellence & Affiliate members and the formation of the Batten Disease Global Research Initiative during Saturday night’s Dinner Dance at [...]
PHOTO GALLERY | 2024 Annual Family Conference
Go behind the scenes of the 2024 BDSRA Foundation Annual Family Conference in St. Louis, MO with this photo gallery produced by Ira Graham III Photography & Films. Pre-Conference Day - Thursday, July 11, [...]
SURVEY: Batten Disease Research Priorities
We are now leading phase 2 of the Batten disease research priority-setting process in partnership with Kennedy Krieger Institute. A summary list of research questions about Batten disease was created using the responses from [...]
JUNE NEWSLETTER | Read The Illuminator
June 2024 [...]
