DoD Duchenne Muscular Dystrophy Investigator-Initiated Research Award

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The DMDRP Investigator-Initiated Research Award (IIRA) mechanism was first offered in FY11. Since then, 40 IIRA applications have been received, and 7 have been recommended for funding. The DMDRP IIRA supports translational research that will accelerate the movement of promising ideas in DMD into clinical applications. Translational research may be defined as an integration of basic science and clinical observations with the specific goal of developing new therapies. While the ultimate goal of translational research is to move an observation forward into clinical application, translational research is most effective as a two-way continuum between the bench and the bedside. Within this continuum, the IIRA supports mid-stage or later translational research projects, including early-phase, proof-of-principle clinical trials and DoD FY13 DMDRP Investigator-Initiated Research Award 4 correlative studies to better inform the development of drugs, devices, and other DMD interventions. Research projects may also include preclinical studies in animal models, human subjects, and human anatomical substances. All projects should adhere to a core set of reporting standards for rigorous study design. The standards are described fully in www.nature.com/nature/journal/v490/n7419/full/nature11556.html. While these standards are written for preclinical studies, the basic principles of randomization, blinding, sample-size estimation, and data handling derive from well-established best practices in clinical studies and should be applied to those projects as well. Studies proposed under this award mechanism should not include: • Target discovery • Drug screening • Mechanism of action studies • Hypothesis-driven pathophysiology studies Applications must include preliminary data that are relevant to DMD and the proposed project. Clinical trials are supported by this award mechanism. Optional Qualified Collaborator: The FY13 DMDRP strongly supports collaborative research between laboratory scientists and clinical researchers, and between academic scientists and biotechnology/pharmaceutical industry scientists. Collaborations that bring new perspectives from other disciplines, or bring new investigators into the DMD field, are also strongly encouraged.

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