Pilot Projects of the LYSOSOMAL DISEASE NETWORK (LDN)

The Lysosomal Disease Network (LDN), a consortium organized under the NIH Rare Diseases Clinical Research Network program, aims to improve the treatment of lysosomal diseases by learning more about each of these conditions by funding a variety of projects including 1-year pilot studies. LDN intends to fund 1-2 pilot projects, on an overlapping annual basis, that promote innovative research at the forefront of new therapies/technologies/advancements for lysosomal disease. Pilot studies must be clinical in nature (either through the use of human subjects or human specimens) and push forward the overarching themes of the LDN: advances in clinical trial readiness, newborn screening, long-term outcomes, and global reach.

Potentially fundable areas include, but are not limited to:

  • Research that gathers data for feasibility, potential, and efficacy of new biomarkers for monitoring disease progression and treatment outcomes in lysosomal diseases.
  • Research projects that provide a foundation for natural history studies for under-researched lysosomal diseases using the expertise of the LDN cores and data already generated.
  • Development of new clinical methods and instruments.
  • Demonstration of efficacy for a method or approach leading to a clinical trial proof-of-concept and/or a phase I trial.

At the conclusion of the funding period, July 30, 2022, the investigator must provide (a) a brief summary of the results and (b) written plan for a peer-reviewed publication that acknowledges support of the NIH-funded Lysosomal Disease Network using the NIH-stipulated format.

  • Due date: June 15, 2021. Incomplete or late applications will not be considered.
  • Decision date: July 25, 2021
  • Funding period: August 1st, 2021 – July 30, 2022
  • Applications must be on NIH 398 (http://grants.nih.gov/grants/funding/phs398/phs398.html) forms and adhere to the page limits listed below. Biosketches and budgets must be included.
  • To be eligible, the applicant must be able to receive this funding through grant/contract from the University of Minnesota through an academic institution or medical center that is actively receiving other funding from the U.S. National Institutes of Health.
  • Applications must include the following items on NIH 398 pages (with these strict page limits). Applications which are not in this format, do not adhere to these page limits, or are incomplete, or arrive after the June 15, 2021 deadline will not be considered.
    1. Abstract (up to 360 words)
    2. Specific Aims (1 page)
    3. Research Strategy (Background and Significance, Innovation, Approach (maximum 6 pages)
    4. NIH-style Biographical Sketch for PI and any other Key Personnel (maximum of 5 pages each)
    5. Budget (1 page) and Budget Justification (1 page).
    6. Submit applications by midnight June 15, 2021 to Brenda Diethelm-Okita (dieth001@nullumn.edu).

Qualified individuals from under-represented groups and individuals with disabilities, who are in health-related sciences, are encouraged to apply.