Batten Disease Research and Treatment

BDSRA has funded 7 million dollars in scientific research across all forms of Batten disease that leveraged millions more in federally funded research grants. Each year, projects funded are announced at our Family Conference and published on our website. To view this year’s research focus and to learn about our research efforts in greater detail visit the  section of our website.

No specific treatment is known that can reverse the symptoms of any form of Batten disease.  For most forms, there are no treatments available that can slow or stop disease progression.  However, in 2017 the FDA approved an enzyme replacement therapy for CLN2 disease (TTP1 deficiency) called cerliponase alfa (Brineura) that has been shown to slow or halt the progression of symptoms.

Seizures can sometimes be reduced or controlled with anticonvulsant drugs.  Other medicines are available to treat anxiety, depression, parkinsonism, and spasticity.  Additional medical problems can be treated appropriately as they arise.  Physical and occupational therapy may help those with the disease retain function as long as possible.  Support groups can help affected children, adults, and families to share common concerns and experiences, and to cope with the severe symptoms of the disease.

BDSRA is involved in many other aspects other than funding research. We facilitate progress in the areas of scientific research and advocacy by collaborating in the global Batten disease community to create the building blocks that make clinical trials possible and we often serve as the Advocate and voice of the patient in the drug development process.

We are always happy to talk to anyone who has questions about the process or if you would like to make a contribution to our program to fund research please contact us at info@bdsra.org anytime.

Research_Treatments_Kielian and Olivia

What Next?

Newly Diagnosed

If you are a newly diagnosed family, we welcome you to connect with BDSRA further and request a new family packet with additional information and resources available.

Family Support Services

We offer direct support services, guidance navigating difficult systems and can help you connect to other families and experts in the field.

Annual Family Conference

Hundreds of family members, children and researchers gather each year to learn about patient care, education, and the latest in science.

Help Our Mission

Whether its advocacy or financial support, there are many ways to join us and strengthen our community working together toward a common goal.

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