Latest News

CLN2 Enzyme Replacement Trial Opens First US Site


We are happy to announce that the first United States site for the BioMarin CLN2 enzyme replacement study has been listed on clinicaltrials.gov. Dr. Emily De los Reyes at Nationwide Children’s Hospital in Columbus and her co-PI, Dr. Lenore Leowald are currently assessing children to begin the study in December. To learn more about the official guidance, and criteria, go to http://1.usa.gov/1Hr3IbITracy VanHoutan, Lance Johnston, researchers from universities all over the world and BioMarin have made this important advancement for families and children with our utmost thanks!

BDSRA Opens RLOI for 2014 Funding Cycle


BDSRA Opens RLOI for 2014 Funding Cycle

COLUMBUS, Ohio, October 2, 2014 — The Batten Disease Support and Research Association (BDSRA) has issued a request for letters of intent (RLOI) for the 2014 research grant cycle.   BDSRA supports scientific investigations through an annual merit review process, awarding grants to researchers throughout the world.

For the current call for LOIs, BDSRA seeks innovative research projects that have the potential to advance therapeutic strategies for all or any of the Neuronal Ceroid Lipofuscinoses.  Each award, depending on funding availability, will be no more than $60,000 for a one-year period.

Letters of intent must be submitted by November 15, 2014.  The LOIs will be reviewed by members of the BDSRA board and invited scientific reviewers.  Requests for full proposals that will be peer reviewed will be made in February, 2015.

The LOI is limited to two pages and must describe the proposed Batten disease research project, including aims, rationale, methodology, statement of translation, and a full budget.  The applicant’s biographical sketch (NIH Form PHS 398) must be attached.  Awards will be announced mid-year.

To submit a LOI, applicants need to go to: https://proposalcentral.altum.com/default.asp?GMID=125

BDSRA is the largest nonprofit organization in North America dedicated to funding research, advancing education, and providing family support services.  As a rare, fatal, inherited disorder of the nervous system, Batten disease has no known treatment or cure.  Private funding for Batten disease research has advanced through partnerships of BDSRA with family foundations and European Batten organizations. Co-funders with BDSRA for research projects in 2012 and 2013 included the Batten Disease Family Association of the United Kingdom, BDSRA-Australia, Noah’s Hope, Hope 4 Bridget, NCL-Stiftung, Bee for Batten Ireland, Beyond Batten Disease Foundation, and BioMarin.

For additional information contact info@bdsra.org or go to www.bdsra.org.

The Fall 2014 BDSRA Newsletter is here


For conference highlights, board news, personal stories and research updates click here Fall 2014 BDSRA Newsletter

2013 BDSRA Annual Report


Read about research strategy, family stories, grant awards and how donors make a difference to carry out the mission of BDSRA here

International Partners Advance Batten Disease Research Targets


BDSRA, in collaboration with international and family foundation partners,
has announced grants totaling $408,000 to advance research in Batten disease.


 International Funding Partners Advance Batten Disease Research Targets  

COLUMBUS, OHIO (April 24, 2014)  The Batten Disease Support and Research Association (BDSRA), in collaboration with international and family foundation  partners, has announced grants totaling $408,000 to advance the development of treatments and cures for Batten disease.

Co-funders BDSRA-Australia, the Batten Disease Family Association of the United Kingdom, Noah’s Hope, and Hope 4 Bridget joined with BDSRA to support projects that span the United States, the United Kingdom, Australia, and Germany.   Investigations to identify drug targets, drug discovery, and expand a patient registry have been funded through awards to six researchers:  David Palmer, PhD, Lincoln University of New Zealand; Stephan Storch, PhD, University Medical Center Hamburg-Eppendorf, Germany; Angela Schulz, PhD, University Medical Center Hamburg-Eppendorf, Germany; Jonathan Cooper, PhD, King’s College, London; Jeffery Gerst, PhD, Weizmann Institute of Science, Israel; and Erika Augustine, MD, the University of Rochester.

The DEM-CHILD NCL Patient Database project led by Schulz is the first worldwide attempt to collect and analyze clinical data of all NCL forms through a consortium of NCL clinicians from 11 countries.  Expanding a worldwide patient registry for Batten disease is a BDSRA strategic funding priority because of its necessity to understand the disease, identify novel biomarkers, advance clinical trials, and develop effective therapies.

With an emphasis on cutting-edge scientific inquiries to build on existing investigations in gene therapy, enzyme replacement, and drug discovery, BDSRA continues to identify and fund research with the highest potential to formulate treatments.

BDSRA and its co-funders select grant recipients through a scientific merit review process in which proposals are evaluated according to standards that screen significance of the project to advance NCL research, strategy to accomplish objectives, and potential for translation to treatments and cures.

As a rare, fatal, inherited disorder of the nervous system, Batten disease has no known treatment or cure. Batten disease is the most common, inherited neurodegenerative disorder found in children, and is also known as a group of disorders called Neuronal Ceroid Lipofuscinosis (NCL).  There are multiple forms of the disease, which is most commonly diagnosed between infancy and school age.

BDSRA is the major support and research organization in North America for Batten disease.  The first research grant from BDSRA was awarded in 1992, and since then more than $7.4 million has been allocated toward the search for treatments and cures.  The organization is the largest provider globally for Batten disease patient and family support, education, research, and awareness programs.


Grants and Patient Registry to Advance Batten Disease Research


Read more about the DEM-CHILD project that is a funding priority for BDSRA to expand a critical patient registry and details about the research projects and grants awarded for the 2013 RFP program.  Go to 2013 RFP Grants

April 2014 Newsletter

BDSRA announces global research grants and programs in April Newsletter.  In collaboration with international and family foundation partners, BDSRA has awarded research grants for 2013 that span the U.S., Australia, Germany, and the United Kingdom.  Proposals were funded that focused on identifying drug targets and drug discovery, developing a patient registry, and expanding projects in the basic biology of Batten disease.  To read more details as well asnews about programs, conference, and families, go to April Newsletter

ourboys 5k/10k


ourboys 5k/10k
April 12, 2014
Harris Road Middle School
1251 Patriot Plantation Blvd
Concord NC 28027

Online Registration and more info at http://davidsontiming.com/2013/11/our-boys-5k-10k-2014/

Run the Creek 5K


Run the Creek 5k
March 22, 2014
9:00 am
Highland Creek Elementary
7242 Highland Creek Parkway
Charlotte NC 28269

Online Registration and more info at http://jonesracingcompany.com/run-the-creek-5k/

Get Involved and Make it Happen: February is membership month for BDSRA


To join or renew your 2014 annual membership,
which is $40 per family, go to 2014 Membership Dues

Why Join BDSRA?  Because membership is a vital link to others
facing Batten disease, and membership strength is a leading indicator
to policy makers and pharma that Batten disease deserves
attention, support, and funding.  Read more about membership here

For questions email tkirby@bdsra.org