Cure Sanfilippo Foundation: Pilot Funding Mechanism

Overview

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.

Funding Mechanisms

Cure Sanfilippo Foundation is pleased to announce an open call for research proposals.

Proposals will be considered under the following mechanisms:

* Pilot Awards
* ADVANCE Awards

All funding awards are contingent upon alignment with the Foundation’s goals and priorities as informed by recognized gaps in clinical care and/or scientific knowledge 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. Award funding is milestone driven and proposals should be written with clear milestones.

Pilot Funding Mechanism

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

Year 2 funding will be dependent upon progress demonstrated in Year 1. Funding is milestone driven.

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.

Eligibility

_You can learn more about this opportunity by visiting the funder's website._

* Who can apply:
* Academic investigators
* For-profit entity investigators