Funding Opportunities for Sanfilippo Research

Interested in Applying for Funding to Research Sanfilippo Syndrome?

Are you a researcher who is interested in collaborating with and/or applying for research funding from Cure Sanfilippo Foundation to study Sanfilippo syndrome?

Cure Sanfilippo Foundation seeks to support research that fills critical gaps in current knowledge and access to treatment across basic and translational science, clinical care, and therapeutic interventions in clinical trials. Our overarching goal is to advance a pipeline of scientific discovery which will improve the lives of children with Sanfilippo syndrome. Scientist-clinician-patient collaborations are highly encouraged and an area of active engagement for the Foundation.

Dissemination of research findings through publications and presentations are required responsibilities of funded applicants. Sharing of data for broad scientific advancement is especially critical in the context of ultra-rare diseases like Sanfilippo syndromes. Applicants are to include a data sharing plan and informed consent language which enables data/biological sample sharing for all clinical study proposals. You may also learn more about the Foundation’s efforts around data-sharing.

Cure Sanfilippo Funding Mechanisms

The next call for Letters of Intent is anticipated to open in late 2024/Jan 2025. More details on the timing will be updated here as they become available.

All funding awards are contingent upon alignment with the Foundation’s goals and priorities as informed by recognized gaps in scientific knowledge or clinical care which present significant barriers to the development of beneficial therapies and improved quality of life for affected individuals. Funding levels and award categories may vary over time to adapt to the ongoing funding commitments by the Foundation and availability of resources.

At this time we anticipate the next opportunity will include the following funding mechanisms:

  • Pilot Awards
  • ADVANCE Awards
  • (New applications for Fellow Awards will be deferred this round due to carry over renewal from the last granting period. Future opportunities are anticipated.)

Priority areas of investigation: details pending

  • A focus on clinical studies is anticipated.
  • Novel strategies and investigations outside of the priority areas will also be entertained.

How to Apply

The next call for Letters of Intent is anticipated to open in late 2024/January 2025. Details will be updated here as they become available.

Sample forms are available as a reference to provide you insights into past requirements, allowing you to better prepare for an upcoming application. Future forms may vary.

If you would like to consult with the Foundation’s Chief Science Officer about your research ideas, we welcome you to reach out regardless of grant round timing. Please email Dr. O’Neill at

Details of Previous Funding Mechanisms

Previous Priority Areas of Investigation

  • Disease/treatment impacts and pathology of the gastrointestinal system (inclusive of the enteric nervous system)
  • Disease/treatment impacts and pathology of the peripheral nervous system and/or spinal cord
    Personalized medicine and framework mechanisms, inclusive of n of 1 approaches
  • Evaluation of gait and mobility assessments over the lifespan, fit for use in clinical trials and community settings
  • Characterization of movement disorders in patients with Sanfilippo and/or an appropriate animal model, exploration of therapeutics, emphasis on cross disease collaborations

Exploration of the measurement of these disease impacts/outcomes in patients using clinically applicable tools and tests will be viewed favorably. Projects outside of the priority areas will also be entertained.

General Funding Mechanisms

Pilot Funding Mechanism

$50,000 USD per year (total costs), up to two years

Year 2 funding will be dependent upon progress demonstrated in Year 1

ADVANCE Sanfilippo Funding Mechanism

$50,000 USD or more per year (total costs)

This funding opportunity is intend for proposals with a larger scope of work and funding needs

Fellowship Award Mechanism

$60,000 USD per year (total costs), for up to two years

This award is highly competitive and need-based.

The Foundation’s aim in supporting trainees is to build a pipeline of talent among early career scientists and clinicians focused on Sanfilippo syndrome from within and outside of the field of lysosomal diseases.

A Letter of Intent is not required in order to apply.

For Questions

If you have questions about the Foundation’s funding opportunities through Cure Sanfilippo Foundation, please email us at