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What We Are Funding

Today we find ourselves poised at an incredible crossroads in Duchenne. We are proud of our investments in research and that the Duchenne pipeline is so rich with promise. In total, PPMD has invested over $45 million into Duchenne research which has leveraged over $500 million in additional funding.

PPMD continues to drive change in the research arena. Our grant cycles offer opportunities to investigators and help us keep our finger on the pulse of research. PPMD’s Scientific Advisory Board expertly reviews every grant that comes in and offers funding or advice on how to proceed to garner funding in the future. Our collaboration with academic research institutes and industry partners helps us push potential treatments to clinic faster, which in turn provides access to the Duchenne community and patients faster.

We don’t just look at one problem, or one bit of research, but rather we are looking comprehensively at the entire landscape and trying to intercept areas where time could be lost and opportunities missed. We invest in research that will impact every person touched by Duchenne, no matter their age, stage of progression, or mutation.

Our Funding Portfolio highlights new treatments and therapies at various stages of development. Your support can turn their promise into success.


Current Projects Needing Funding


2015 Duchenne Regulatory Science Consortium Research Agreement Stephen Broadbent, Critical Path Institute Research Grant $900,000
2015 Development of VBP15 for Duchenne Muscular Dystrophy Dr. Eric Hoffman, Reveragen Xcelerate Grant $750,000
2015 TREAT-NMD Advisory Committee for Therapeutics (TACT), 11th Meeting, Dublin, Ireland, 27th -29th March 2015 Dr. Volker Straub, University of Newcastle upon Tyne Meeting Grant $14,000
2015 The Research Institute at Nationwide Children's Hospital Myology Course Dr. Kevin Flanigan, University of Utah Meeting Grant $30,000
2015 Clinical trials support for CAT-1004 Dr. Maria Mancini, Catabasis Pharmaceuticals Research Grant $100,000
2015 Exploring the possibility of SERCA2a therapy in DMD dogs Dr. Dongsheng Duan, University of Missouri Exploratory Grant $55,000
2015 Evaluation of exon skipping enhancers for DMD Dr. Carrie Miceli, University of California, Los Angeles Exploratory Grant $50,000
2014 Development of Online Course: PS121 “Disease Mechanisms and Therapies Dr. Rachelle Crosbie-Watson, University of California, Los Angeles Supplement $157,500
2014 A phase 1b open-label, single and multiple ascending dose study ot evaluate the safety, tolerability and pharmacokinetics of HT-100 in patients with Duchenne muscular dystrophy, and a phase 2a extension study Dr. Marc Blaustein,Akashi Therapeutics, Inc. GIFTED $500,000
2014 Predicting the benefit vs the side effects of corticosteroids in Duchenne muscular dystrophy Dr. Robert Griggs, University of Rochester Research Grant $78,357


View our full Funding Portfolio



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Funding Portfolio
Programs & Priorities
Review Committees 

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