TUCSON, Ariz. August 6, 2026 — Critical Path Institute® (C-Path) today announced it has been awarded a grant in the amount of $1.9 million under the Department of Defense Duchenne Muscular Dystrophy Research Program in partnership with Monroe Carell Jr. Children’s Hospital at Vanderbilt. The grant is administered by the Office of the Congressionally Directed Medical Research Programs.
The funded project, co-led by C-Path’s Duchenne Regulatory Science Consortium, is titled “Characterizing Disease Progression in Duchenne Muscular Dystrophy-associated Cardiomyopathy” and aims to deepen scientific understanding of how heart disease develops and progresses in individuals living with Duchenne muscular dystrophy (DMD).
C-Path’s D-RSC Executive Director, Ramona Belfiore-Oshan, Ph.D., will serve as the partnering principal investigator of the project together with Jonathan Soslow, M.D., of Vanderbilt Health, a collaboration that reflects a strong, multi-institutional effort to address urgent unmet needs in Duchenne research.
DMD is a fatal disease with limited therapeutic options and no cure, and while cardiomyopathy affects nearly all individuals living with the condition, and is the leading cause of death, it remains understudied and difficult to measure. Existing tools often fail to detect early cardiac disease or predict its progression, limiting the development of effective therapies.
This project will integrate diverse datasets and advanced quantitative approaches to better define the natural history of cardiomyopathy in Duchenne, identify key biomarkers, and support the development of drug development tools that can optimize the design and conduct of clinical trials for candidate drugs. These outcomes are expected to contribute significantly to the broader DMD research ecosystem and inform regulatory decision-making.
Importantly, these advances may extend beyond Duchenne, supporting cardiac drug development across other neuromuscular diseases. Ultimately, this work aims to remove key barriers to clinical trials and drive faster progress toward disease-modifying treatments.
Building on C-Path’s proven expertise in disease modeling and trial simulation, the effort will help enable more efficient, data-driven drug development. Together, C-Path’s Duchenne consortium and its collaborators bring expertise in clinical care, data science, cardiac imaging, regulatory science and patient advocacy to create a robust framework for analyzing disease progression in Duchenne-associated cardiomyopathy.
“This award represents an important step forward in our collective effort to understand and ultimately improve cardiac measures for individuals living with Duchenne,” said Belfiore-Oshan. “By leveraging the collaborative infrastructure of C-Path’s Duchenne consortium, we aim to generate high-quality, regulatory-ready evidence that can inform clinical trials and accelerate the development of effective therapies targeting cardiac disease in DMD.”
“The DMDCCC is excited to partner with C-Path,” said Soslow. “They have been a leader in the neuromuscular field for many years and their modeling has played a significant role in the development of successful clinical trials for DMD skeletal muscle therapeutics. Currently, there are no DMD-specific therapies approved for cardiovascular disease, despite it being the leading cause of mortality in DMD. We are hopeful that this collaboration will lead to similar advances in DMD cardiac modeling and help investigators craft clinical trials that lead to novel DMD cardiovascular therapies.”
“For more than two decades, C-Path has brought together scientists, regulators, patient communities and industry to solve problems that no single organization could tackle alone, and this project is a powerful example of that mission in action,” said C-Path Chief Executive Officer Klaus Romero, M.D., M.S., FCP. “Cardiomyopathy remains one of the most serious, yet understudied threats facing individuals living with Duchenne muscular dystrophy, and by generating the regulatory-ready evidence needed to understand and measure it, we hope to move the field closer to therapies that can truly change the trajectory of this disease. We are deeply grateful to the Department of Defense for its support, and we remain committed to the individuals and families affected by Duchenne muscular dystrophy, who inspire this work every day.”
C-Path’s Duchenne Regulatory Science Consortium is a public-private partnership dedicated to advancing regulatory science tools and accelerating therapy development for Duchenne muscular dystrophy. By fostering collaboration across academia, industry, patient advocacy groups, and regulatory agencies, the consortium plays a critical role in shaping the future of Duchenne research.
About the Critical Path Institute
Critical Path Institute® (C-Path) is an independent, nonprofit organization established in 2005 as a public-private partnership in response to the FDA’s Critical Path Initiative. C-Path’s mission is to lead collaborations that advance better treatments for people worldwide. Globally recognized as a pioneer in accelerating drug development, C-Path has established numerous international consortia, programs and initiatives that currently include more than 1,600 scientists and representatives from government and regulatory agencies, academia, patient organizations, disease foundations and pharmaceutical and biotech companies. With dedicated team members located throughout the world, C-Path’s global headquarters is in Tucson, Arizona, C-Path’s Europe subsidiary is headquartered in Amsterdam, Netherlands. For more information, visit c-path.org.
Critical Path Institute is supported by the Food and Drug Administration (FDA) of the Department of Health and Human Services (HHS) and is 56% funded by the FDA/HHS, totaling $23,740,424, and 44% funded by non-government source(s), totaling $18,881,611. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government.
Media Contacts:
Roxan Triolo Olivas
C-Path
520.954.1634
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C-Path
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