The Clinical Translational Science Institute at the University of Florida has awarded a nearly $20,000 grant to Sarah Kim, Ph.D., a research assistant professor in the UF College of Pharmacy, to study potential therapies of Duchenne muscular dystrophy, or DMD. Kim will lead a research team charged with developing a mathematical model-based clinical trial simulation tool using magnetic resonance imaging and spectroscopy data of patients with DMD collected through the ImagingDMD project at UF. The tool will inform optimizing clinical trial designs of efficacy evaluation studies of potential therapies for DMD.
“The interdisciplinary model-based approach could be used as an example to inform drug discovery and development pipelines through simulations for DMD and other diseases,” Kim said.
Stephan Schmidt, Ph.D., F.C.P., an associate professor of pharmcaceutics, the director of the Center for Pharmacometrics and Systems Pharmacology in the UF College of Pharmacy and the Certara Endowed Professor, will serve as a mentor on the grant, along with Krista Vandenborne, P.T., Ph.D., a distinguished professor and chair of the department of physical therapy and director of the ImagingDMD project. Rebecca Willcocks, Ph.D., a research assistant professor in the department of physical therapy is serving as a co-investigator on the grant.
Duchenne muscular dystrophy is a genetic disease and progressive form of muscular dystrophy. It primarily occurs in males and is often diagnosed in young children before age five. Early symptoms may include difficulty sitting, standing, walking and learning to speak. DMD is caused by a genetic mutation that prevents the body from producing a protein needed to support muscle movement.