Parent Project Muscular Dystrophy Awards First End Duchenne Grant
Brown University to Receive Grant Promoting Translational Research in Rare Muscular Disease
The End Duchenne Grant Award Program was created by PPMD in partnership with the National Institutes of Health (NIH) in an effort to ensure continuation of promising Duchenne research and translation to human studies. The End Duchenne Grant Award Program is a bridge grant provided by PPMD to selected research projects that receive scores beyond the current funding paylines of the NIH Institutes and Centers supporting Duchenne research.
Explains Ms. Furlong, “With its research budget growing tighter each year, the NIH can fund only a fraction of the many promising applications it receives. Currently, only those grants which score in the top percentiles are able to be funded.”
The End Duchenne Grant Award Program represents the first Duchenne specific bridging program and the first ongoing bridge funding program to be presented in the rare disease category. As a collaborative effort with the NIH, the selection strategy behind this award is very different than any other private research investment in Duchenne, yet the goal of this award remains consistent with all of PPMD’s research initiatives: to advance promising research which will impact this generation affected by Duchenne.
The recipient of an End Duchenne Grant must be focused on translational research (the process of applying ideas, insights and discoveries generated through basic scientific inquiry to the treatment or prevention of disease or injury). The NIH will notify investigators whose translation grant applications score well, but not within the NIH funding levels, to submit their applications and score sheets to PPMD. Ms. Furlong continues, “PPMD’s Scientific Review Board will re-review these applications seeking to identify those with the greatest potential to ultimately help all boys with Duchenne.”
The End Duchenne Grant Award Program was designed to enable investigators to continue their projects and generate additional data for a successful re-application within a 12 to 24 month period, thus leveraging additional Duchenne-specific research dollars.
Ms. Furlong is thrilled that PPMD was able to launch the grant program, and is particularly excited that
When asked what these grant funds will do for the University’s research, Dr. Fallon responded, “Our lab is pioneering a novel DMD therapeutic based upon the systemic delivery of the extracellular matrix protein rhBGN (recombinant human biglycan). Several characteristics of rhBGN suggest that it could be an effective therapy for Duchenne, including that rhBGN counters dystrophic pathology and improves muscle function. This is the most important finding we have made in terms of Duchenne relevance and given the lack of effective therapies for Duchenne, this finding alone is motivation for pursuing this agent as a potential therapy. The End Duchenne Grant will help us continue our pursuit and give us the data we need to receive further funding from the NIH next year. We couldn’t be more honored to receive this award and more importantly, receive the faith of the PPMD community.”
Duchenne, the most common form of childhood muscular dystrophy, is a progressive and fatal muscle disorder affecting boys and young men that causes the loss of muscle function, wheelchair dependency and a decline in respiratory and cardiac function.
An applicant seeking support from the End Duchenne Grant Award 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 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. For more information or to apply, please visit www.parentprojectmd.org/EndDuchenneGrant.
Parent Project Muscular Dystrophy (PPMD) is a national not-for-profit organization founded in 1994 by parents of children with Duchenne and Becker muscular dystrophy. The organization’s mission is to improve the treatment, quality of life and long-term outlook for all individuals affected by Duchenne muscular dystrophy through research, advocacy, education and compassion. PPMD is headquartered in
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SOURCE Parent Project Muscular Dystrophy