Resources

As the rare disease community strives to better understand Batten disease, consider these tools and partner sites for continued learning about life with this disease.

Batten Disease Clinical Trials

There is a growing body of research on Batten Disease and the CLN5 and CLN7 subtypes in particular, as well as the various CLN7 or CLN5 gene variants that cause the disease. This research is intended to fuel advances for the Batten patient and healthcare community.

There are a limited number of trials related to Batten disease. All current clinical trials are listed on ClinicalTrials.gov.

Get to Know the Batten Disease Community

Additional Resources

Batten Disease

BDSRA is the largest support and research organization dedicated to Batten disease in North America. BDSRA is dedicated to funding research for treatments and cures, providing family support services, advancing education, raising awareness, and advocating for legislative action.

https://www.bdsra.org/

BDFA

The Batten Disease Family Association (BDFA) is a UK non-profit that aims to support families, raise awareness, and facilitate research into the group of devastating neurodegenerative diseases commonly known as Batten disease.

http://www.bdfa-uk.org.uk/

Bee For Battens

Bee For Battens, as part of the Saoirse Foundation is a place where families and persons affected by Battens Disease can come together to help raise awareness about this devastating condition whilst providing support for one another. Our contacts and networking with other Battens Disease parents, families & Charities around the globe has grown and grown, which has provided us with the most reliable source of information and support available, from those who live with Battens day in day out.

https://www.beeforbattens.org/

Rare Disease Network

Rare Diseases Clinical Research Network is a group committed to advancing medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment, and data sharing.

https://www.rarediseasesnetwork.org/

Global Genes

Global Genes is an organization that helps to build awareness, educate the global community, and provide critical connections and resources that equip advocates to become activists for their rare diseases.

www.globalgenes.org

The National Organization for Rare Disorders

The National Organization for Rare Disorders (NORD®), an independent nonprofit, is leading the fight to improve the lives of rare disease patients and families. We work together to accelerate research, raise awareness, provide valuable information and drive public policy that benefits the estimated 25-30 million Americans impacted by rare diseases.

www.rarediseases.org

EveryLife Foundation

EveryLife Foundation is dedicated to advancing the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy.

www.everylifefoundation.org

Eurordis

EURORDIS-Rare Diseases Europe is a non-profit alliance of 864 rare disease patient organizations from 70 countries that work together to improve the lives of the 30 million people living with rare disease in Europe.

https://www.eurordis.org/

Genetic Alliance

Genetic Alliance is an advocacy organization that engages individuals, families, and communities to transform health.

www.geneticalliance.org

Child Neurology Foundation

The Child Neurology Foundation connects partners from all areas of the child neurology community so those navigating the journey of disease diagnosis, management, and care have the ongoing support of those dedicated to treatments and cures.

https://www.childneurologyfoundation.org/

Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine is an international community of small and large companies, non-profit research institutions, patient organizations, and other sector stakeholders dedicated to realizing the promise of regenerative medicine for patients around the world.

https://alliancerm.org/

ARM Foundation

The ARM Foundation for Cell and Gene Medicine educates, engages, and empowers patients, caregivers, industry leaders, and other stakeholders to help advance the science and benefits of gene and cell therapy.

www.thearmfoundation.org

The Mighty

The Mighty is a supportive health community for people facing challenges and their families.

www.themighty.com

National Alliance for Caregiving

Established in 1996, the National Alliance for Caregiving is a non-profit coalition of national organizations focusing on advancing family caregiving through research, innovation, and advocacy. The Alliance conducts research, does policy analysis, develops national best-practice programs, and works to increase public awareness of family caregiving issues. Recognizing that family caregivers provide important societal and financial contributions toward maintaining the well-being of those they care for, the Alliance supports a network of more than 80 state and local caregiving coalitions and serves as Founder and Secretariat for the International Alliance of Carer Organizations (IACO).

https://www.caregiving.org/

Medical articles published about Batten Disease (CLN5/7) can be found below

Wheeler, R. B., Sharp, J. D., Mitchell, W. A., Bate, S. L., Williams, R. E., Lake. B. D., & Gardiner, R. M. (1999). A New Locus for Variant Late Infantile Neuronal Ceroid Lipofuscinosis—CLN7, Molecular Genetics and Metabolism, 66(4): 337-338. Retrieved August 16, 2019, from link.

Savukoski, M., Klockars, T., Holmberg, V., Santavuori, P., Lander, E., & Peltonen, L. (1998). CLN5, a novel gene encoding a putative transmembrane protein mutated in Finnish variant late infantile neuronal ceroid lipofuscinosis. Nature Genetics, 19: 286-288. Retrieved August 16, 2019, from link.

McBride, J. L., Neuringer, M., Ferguson, B., Kohama, S. G., Tagge, I. L., Zwieg, R. C., Renner, L. M., McGill, T. J., Stoddard, J., Peterson, S., Su, W., Sherman, L. S., Domire, J. S., Ducore, R. M., Colgin, L. M., & Lewis, A. D. (2018, Nov.). Discovery of a CLN7 model of Batten disease in non-human primates, Neurobiology of Disease, 119: 65-78. Retrieved December 17, 2019, from link.