Clinical research aimed at understanding the natural history of rare disorders is critical for advancing medical knowledge and improving patient care. Rare disorders, due to their low prevalence, often lack comprehensive data, making it challenging to develop effective interventions and treatments. This funding opportunity seeks to address this gap by supporting clinical research that meticulously documents the progression, variability, and outcomes of rare diseases over time (i.e., natural history studies). Such research is essential for identifying potential biomarkers, elucidating disease mechanisms, and ultimately guiding the development of targeted therapies.
This funding opportunity supports natural history studies in one or more rare diseases for up to three years at $20,000 direct costs per year. We encourage applications from investigators committed to advancing rare disease research and fostering collaborative, multidisciplinary approaches to uncovering critical insights that will pave the way for future medical breakthroughs.
Requirements
- The principal investigator (PI) must be a faculty member and must agree to join the faculty of the Center for Rare, Undiagnosed & Genetic Disorders (CRUGD)
- The application must partner with one or more patient advocacy groups (PAG). A letter of support is required (in the full application) demonstrating the commitment and role of the PAG in the project. Financial support is highly recommended if feasible.
How to apply
Letters of intent (LOI) are required. Please download and complete the LOI application form (below). LOIs must be returned by email to rarediseases@wustl.edu by 5:00 p.m. Central Time on March 14, 2025. Completed LOIs must include answered questionnaire, NIH style biosketch of the PI(s) and the name and brief description of the PAG and their commitment to the project.
Selected LOIs will be announced by April 4, 2025 and invited to submit a full proposal by 5:00 p.m. Central Time on May 9, 2025. The full submission must include: a lay abstract (250 words, which will be made public in case of project being awarded), specific aims (1 page maximum), research plan (5 pages maximum), references, detailed budget and budget justification, support letter from the collaborating PAG, and biosketch(s) and other support for the PI(s) and co-investigator(s).
Funding announcements will be the week of June 16, 2025. The project funding period starts July 1, 2025.
Timeline
| March 14, 2025 | Letters of intent (LOI) due by 5:00 p.m. Central Time |
| April 4, 2025 | Select applicants will be invited to submit full proposal |
| May 9, 2025 | Full project submissions due by 5:00 p.m. Central Time |
| June 16, 2025 | Awards announced |
| July 1, 2025 | Award start date |