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End Duchenne Grant Award Program

PPMD has developed the End Duchenne Grant Award Program in partnership with the National Institutes of Health (NIH) in an effort to ensure continuation of promising research and translation to human studies. The End Duchenne Grant Award Program is a bridge grant provided by PPMD to promising research projects that receive scores beyond the current funding pay lines of the National Institutes of Health (NIH) Institutes and Centers supporting Duchenne muscular dystrophy research. The goal and hope of this grant program is that bridge funding would accelerate promising research forward and enhance the success of competition for federal dollars toward new treatment development and validated clinical measures for Duchenne muscular dystrophy.

Specific Features of End Duchenne Grant Award Program:

1) Any NIH grant type mechanism is acceptable to this program if the work scope of the research is directly applicable to the development of new treatments and/ or the validation of clinical measures for Duchenne muscular dystrophy. Applications applicable for consideration of bridge funding are either:

a.) translational applications, that are designed to move scientific discoveries at any stage of preclinical development to new therapies 
b.) clinical applications designed to validate biomarkers to clinical functional outcome measures or validate clinical outcome measures to increase the efficiency of determining effectiveness of experimental therapeutics in clinical trials.

Applicants must first contact the End Duchenne Grant Program Coordinator to discuss eligibility. Please contact the End Duchenne Grant Program Coordinator for more information.

2)  Awards are made for up to one year with the expectation that the grantee will submit a revised application to the NIH before the end of the PPMD funded bridge year.

3) Applicants can request up to 50% of the first year costs budget as contained within the NIH application. Indirect/overhead costs are not supported in this program.

4)  PPMD will conduct an initial review of eligibility for grant consideration. Ellibigle applications will be reviewed for scope and merit by the End Duchenne Grant Award Review Committee. The review committee will make recommendations to PPMD for funding of the research proposal for an End Duchenne grant award.

5)  Review of applications for End Duchenne Grant award will occur three times per year to coincide with NIH grant review cycles, following each NIH review cycle, provided applications are identified for this program.

Eligibility for the End Duchenne Grant Award Program:

An applicant seeking support for this program must be employed at a for-profit or non-profit organization or institution and have submitted and received a complete review of an application to the NIH directed toward clinical validation studies of biomarkers or functional outcomes or translation of research into human clinical studies specific for Duchenne muscular dystrophy. The applicant must have the resources to conduct the proposed research project and the organization/institution must have appropriate grant administrative capacities for the handling and disbursing of research funds. Applicants are required to contact the End Duchenne Grant Program Coordinator, to discuss eligibility prior to submitting proposals. 

 

Application Process:

The End Duchenne Grant Award Program process builds from the NIH grant review process.

Eligible applicants are requested to provide the following:

1) A proposal to PPMD for funding:

a. Introduction: narrative describing the significance of the proposal to PPMD, outlining the specific work scope and approach-what specific aims (experimentations) would be performed and the rationale for choice of experiments to address issues identified in NIH review and to advance progress of the project, keeping within the budget frame of 50% funding of NIH grant first year direct costs
b. Research methods: Provide details of methods for any experiments not already outlined in NIH grant proposal. New experimentation may be proposed to address issues raised in NIH grant review.
c. Facilities/new personnel (CV) if any additions made from NIH application
d. Anticipated time frame for submission of revised grant application to NIH
e. Budget for proposed research (up to 50% direct costs of first year NIH grant)
f. Statement of commitment: Regarding progress reports, publications and citations, status of NIH grant resubmission, and public relations

2) The NIH grant application for consideration of an End Duchenne Grant Award (required)

3) The NIH Summary Statement to the grant application (required) **If the summary statement is not available prior to submission, contact End Duchenne Grant Program Coordinator to discuss

4) A response to NIH reviewers comments/concerns (optional)

5) Appendix materials (optional)

Click here for an example of the application content (check list). More details regarding the submission of the application will be provided by the End Duchenne Grant Program Coordinator.

Application submission dates

August 25
December 20
April 20

Number of awards each cycle is dependent on the number of applications, outcome of review and the availability of funds. PPMD is not obligated to make awards following each grant cycle.

Review process:

The review of the End Duchenne Grant Award Program addresses these key issues:

1) Significance: Does the proposal meet the objectives of the End Duchenne Grant Award Program to facilitate promising research towards new therapeutics or new clinical validated measures for Duchenne muscular dystrophy? Does the proposal address an approach to developing a new treatment strategy that is not currently being investigated? If a clinical application, does the proposal address the development of new clinical research validation studies that would enhance the efficiency, feasibility or well-being of individuals in Duchenne clinical treatment trials/studies?

2) Scientific merit of the research plan: Given the NIH review and response of the investigator, is the work scope proposed in the PPMD application reasonable and methods sound? Would successful completion of the proposal to PPMD address the issues of the NIH review and enhance success in resubmission? Are there other experiments for the investigator to consider? Would completion of this project advance development towards a new therapeutic or a new clinical validation measures for Duchenne?

3) Investigators/Resources/Environment: in place to perform the work? Are there are any additional issues not identified or addressed with the NIH review and response from the applicant. If the proposal to PPMD contains new specific aims, review any additional personnel and resources for adequacy.

4) Budget-appropriateness for work scope proposed : Does the proposed budget track with the proposed aims?

The review process will maintain strict confidentiality and attend to issues of conflict of interests. Applicants will be informed of disposition of the grant within a short time frame following the review. PPMD will make the final award decisions, in its sole discretion.

For more information on the End Duchenne Grant Award Program, please contact the End Duchenne Grant Program Coordinator.