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"Stronger 2020" from A Foundation Building Strength for Nemaline Myopathy (NM).

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United States
Non-Profit
RFP
Download the rfp file: TU RFP 2019 - GD_TS (1).docx.

A Foundation Building Strength (AFBS) is a non-profit organization that strives to stimulate, advance, and move forward the science of treating NM while supporting the community with information, resources, and solutions. The foundation's primary research goals are to fund and stimulate research into treatment and a potential cure for NM.


"Stronger 2020"

AFBS is excited to announce a new "Stronger 2020" initiative to fund up to $1 million in research grants, advancing our mission to find treatments and solutions for NM. AFBS has and will continue to fund research advancing our understanding of the pathogenesis and potential therapies for NM, as well as devices that support functional and quality of life improvements for NM patients and their caregivers. Under our Stronger 2020 initiative, we are particularly interested in supporting investigators in three main areas although we will consider other creative and highly impactful proposals outside these core areas of focus:


1. Finding Treatments for NM: We seek to fund discovery research of underlying disease mechanisms and preclinical development of potential therapies, as well as the clinical translation of those efforts. We encourage applications aiming at understanding the causes of disease, unraveling pathways involved in disease, identifying novel drug targets or gene therapies, and testing new strategies to treat disease or any of its incapacitating consequences (e.g. contractures, respiratory function decline). We also encourage applications proposing to create or improve disease models (e.g. animal models, patient-derived cell models), biomarkers, or functional outcome measures to assess therapeutic impact.


2.Development of Supportive Care Devices: For this topic, we will consider any device that could improve the quality of life for a patient with NM. This could be an exoskeleton or wheelchair that assists movement, or a device that assists patients with breathing while awake or asleep, or any device that helps patients with any other aspects of NM.


3. Improving Pulmonary Function: Any project that studies or aims to develop products or therapies for the improvement of pulmonary function in NM patients will be considered. This could include clinical studies aiming to investigate methods to improve function and strength of respiratory muscles and/or to optimize clearance of secretions. We are also interested in funding studies or products that improve the measurement and validation of endpoints for pulmonary function. Modalities funded within this topic could include therapeutics, exercise, devices, or platforms for data acquisition and analysis.


To reiterate, research proposals need not be limited to these three areas. AFBS will seriously consider all proposals supporting our mission.


ELIGIBILITY CRITERIA



To be eligible to apply for the Nemaline Myopathy Research Grant, an applicant must:



- Be qualified to conduct a program of original research. Generally this will mean the Principal Investigator holds a Doctor of Medicine (M.D.), Doctor of Philosophy (Ph.D.) or equivalent degree (e.g. D.O.) and has their own laboratory

- Assume both administrative and financial responsibility for the grant

- Have access to institutional resources necessary to conduct the proposed research properly.


There are no geographic restrictions to be eligible.


REQUIREMENTS FOR GRANT APPLICATION



Lay Abstract

Please provide a statement of no more than 300 words explaining the subject of the research proposal, relationship to NM, and its goals. This should be written using relatively simple lay terminology so that an individual without scientific training will understand the statement.


Scientific Abstract

Please provide a statement of no more than 300 words explaining the subject of the research proposal, its relationship to NM, and its goals. This statement will inform the scientific community of the nature of this work.


Research Plan (No longer than 8 pages)

1.Hypothesis and / or Specific Aims: (maximum of one page)

State clearly and concisely the intent of the proposed research and / or the hypothesis to be tested and developed.

2.Background and Significance: (maximum of one page)

Briefly describe the background of the present proposal. Explain the potential significance and relevance of the proposed work to advancing knowledge of NM. Please include a brief description of techniques needed to make the proposed project a success.

3.Preliminary Studies: (maximum of one page)

Show or cite prior work in your laboratory and published papers directly related to the proposed project. Include descriptions of established techniques and collaboration with other scientists.

4.Research Design and Methods: (maximum of two pages)

Provide a detailed discussion of the experimental design and methods to be used to accomplish the specific aims. Please include a detailed timeline for experiments included in the project.

5.Success Criteria: (maximum of two pages)

Provide details of methods used to evaluate the data and prospectively identify criteria to determine success. Identify key milestones that would support moving forward with the original hypothesis and aims or would warrant a revision and update of the original proposal. For quantitative measures, this could include description of anticipated statistical analysis.

6.References (maximum of one page)



All proposals must be submitted through the application portal at buildingstrength.submit.com by 5pm Pacific Time on December 20, 2019.


Team

Provide a detailed description of the team and the roles each team member would play. Include a biosketch for each key investigator (use NIH or similar format).



Budget

Please include a detailed budget and justification that addresses personnel, equipment, supplies, travel, patient care, and any other relevant costs. As a nonprofit, our policy is to not pay indirect cost rates for institutional overhead. We will pay some indirect costs if absolutely required (to be negotiated on a case-by-case basis), but priority will be given to low overhead proposals.


Terms of Award Period

Projects can span more than one year as required by the nature of the research. In general, funding will be tied to milestones. Funding recommendations will be made by the AFBS Scientific Advisory Board and funding decisions will be made by the AFBS Board of Directors.


Amount of Award

"Stronger 2020" awards will total up to $1 million. Multiple grants will be made, and the requested funding should match the proposals. Each proposal can request a maximum of $200,000 for a two-year grant. Well-justified exceptions could be made by email sent to rfp@buildingstrength.org with the subject "Budget Special Request," with at least 2 weeks in advance of the deadline.


Other Policies and Guidelines

Written progress reports and a short video conference to discuss such progress will be required on a quarterly basis for one-year grants or on a semester basis for longer grants. We would like our investigators to publish their results. Regulatory approvals for proposed clinical investigations or animal studies do not have to be in place at the time an application is submitted; however, funding will not commence until all necessary regulatory approvals have been obtained.



Questions? Please reach out to tselucky@buildingstrength.org.

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