Open call for proposals to ADVANCE Sanfilippo syndrome research
Cure Sanfilippo Foundation is now accepting Letters of Interest (LOI) for its ADVANCE Sanfilippo Scientific & Clinical Research Program
Cure Sanfilippo Foundation supports research across basic and translational science, clinical care, and therapeutic interventions, including clinical trials. Our overarching goal is to advance a pipeline of scientific discovery which will improve the lives of individuals with all forms of Sanfilippo syndrome.
Deadline to Apply
Submit LOI using the ProposalCentral platform by 11:00 p.m. ET on March 28, 2025. After review, selected LOIs will be invited to submit a full proposal. ProposalCentral direct link: https://proposalcentral.com/GrantOpportunities.asp?GMID=338
Priority Areas
Priority areas of interest for this submission period include proposals seeking collaborative research with Cure Sanfilippo Foundation to:
- Advance the clinical understanding, diagnosis, measurement tools and management of movement disorders in individuals with Sanfilippo syndromes.
- Evaluate the feasibility of biomarkers related to mitochondrial function, oxidative stress, neurotransmitter profiles, autophagy, mitophagy functions in humans with Sanfilippo syndromes. Preference for clinically-available laboratory tests.
- Increase the evidence base of behavioral therapy (often referred to as Applied Behavioral Analysis) in individuals with Sanfilippo syndromes. May include case series which report on longitudinal data and validated assessment tools.
- Case studies comparing outcomes and/or biomarkers between siblings who have received enzyme restorative therapy vs an untreated sibling.
- Advance innovative translational research for Sanfilippo syndrome which include a plan to begin a clinical trial within 2 years. May include combination therapies. May be subtype specific or basket studies.
Proposals outside these priority areas are also welcome.
Additional Information & Support
More information, including sample forms, are available on Cure Sanfilippo Foundation’s website.
Anyone interested in consulting with the Foundation’s Chief Science Officer about research ideas are welcome to reach out, regardless of grant-round timing, at Research@CureSanfilippoFoundation.org.