DONATE NOW
Research Grant Program 2023-24 Grant Recipients   Previous Research Grants  

BDSRA Australia
Batten Disease Research Grant Programs


Since its formation in 1995, BDSRA Australia has proudly contributed over $1 million toward Batten disease research across Australia, New Zealand, the US, UK and other regions.

In the past five years alone, under the auspices of the competitive BDSRA Australia Research Grant Program, we have invested more than $400,000 toward promising local research right here in Australia and New Zealand. These BDSRA Australia-funded studies have led to groundbreaking discoveries in cell and animal models of Batten disease, helped us better understand some of the key clinical symptoms of Batten disease, and played a pivotal role in advancing a gene therapy to the first clinical trial in humans ( see Funded Projects ).

Our Role in Global Research

In 2024, BDSRA Australia was proud to become an integral part of the global Batten disease research community, as a founding member of the Batten Disease Global Research Initiative (BDGRI). We are honoured to have our Head of Research & Medical Affairs, Dr Ineka Whiteman, appointed as the BDGRI Program Lead.

Together with our partner organisations, BDSRA Australia is working to identify, foster, and collectively invest in the most promising research around the world, to accelerate therapeutic development, improve diagnosis and standards of care, and enhance the quality of life and outcomes for all individuals affected by Batten disease.

Find out more about the Batten Disease Global Research Initiative (BDGRI) here.

Our Research Strategy

Through the Batten Disease Global Research Initiative Grant Program, we aim to invest in the most promising research worldwide that addresses key research questions and areas of unmet need in Batten disease.

This may include, but is not limited to, one or more of the Top Research Priority questions for Batten disease. These Research Priorities were identified through the 2024 Research Priority Setting Partnership survey conducted by the Kennedy Krieger Research Institute in partnership with the BDSRA Foundation, in accordance with the James Lind Alliance Method.

Top Research Priorities for Batten Disease

  1. What are the best gene-targeted therapy options for Batten disease? How can this research be accelerated?
  2. What are valid, reliable biomarkers that can inform Batten disease diagnosis, prognosis or treatment response?
  3. What impact does Batten disease have on organs and body systems outside the central nervous system?
  4. Can disease progression be halted or reversed?
  5. How does Batten disease impact cognition and behavior? What are the best treatment strategies for these symptoms?
  6. What strategies can be used to enhance early diagnosis (including newborn screening)? What are the impacts of early detection and diagnosis?
  7. What interventions are best for maintaining function in Batten disease (including communication, learning, motor skills, mobility, etc)?
  8. Which therapies have the potential for the fastest translation from bench to bedside? How do we accelerate therapy development?
  9. What can be learned about Batten disease from similar lysosomal disorders? Which disease processes represent shared therapeutic targets?
  10. Which complementary health approaches (including diet, supplements, exercise, etc) are helpful for improving symptoms or slowing progression in Batten disease?
  11. What are the common causes and signs of pain and distress in the NCLs, and how do we reliably measure, report, and treat pain and distress?
  12. What are the optimal clinical trial designs for NCLs?
Sign up for the BDGRI Mailing List here
Image