Cure Sanfilippo Foundation: Fellowship Award Mechanism

Overview

_NOTE: This award is highly competitive and need-based. A Letter of Intent is not required in order to apply._

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.

Fellowship Award Mechanism

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.

Funding

$60,000 USD per year (total costs), for up to two years. Renewal application is required for Year 2 funding, if applicable.
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._

* Early career scientists and clinicians focused on Sanfilippo syndrome from within and outside of the field of lysosomal diseases are eligible to apply.