Dec 1, 2020
Local, Structural Scaffold to Stabilize Limb Muscle in Muscular Dystrophy
University of Missouri, School of Medicine
Coulter Biomedical Accelerator
Projects Funded: 2021
Local, Structural Scaffold to Stabilize Limb Muscle in Muscular Dystrophy
Project: Dystrophix Principal Investigators
Clinical PI: Richard Barohn
Executive Vice Chancellor for Health Affairs and Hugh E. and Sarah D. Stephenson Dean, MU School of Medicine
Engineering PI: Steven Segal
Curators Distinguished Professor, Chancellor's Professor of Research Excellence and Margaret Proctor Mulligan Professorship in Medical Research
Engineering Co-PI: Richard Brow (Missouri S&T)
Curator’s Professor of Ceramic Engineering
Aaron Morton, PhD
Medical Pharmacology and Physiology Post-Doctoral Fellow
Abstract: Duchenne Muscular Dystrophy (DMD) is a rare genetic disease that is characterized by progressive muscle degeneration and weakness and is eventually fatal. DMD occurs in 1 in 3500-5000 males born worldwide, and more than 90% of patients become wheelchair-bound by age 15. It primarily affects males, but, in rare cases, can also affect females. The global DMD treatment market is growing at an annual rate of 5% and consists of 290,000 patients paying an average of $2.9B/year for treatment. The major burden facing DMD patients and their families is the decline in mobility from a lack of the gene dystrophin. Dystrophin deficiency leads to fragile muscle fibers that are easily damaged. FDA-approved and pipeline therapies are fraught with drawbacks that include no improvement in muscle function, and secondary consequences of frontline medications. The team is developing a biomimetic micro scaffold (Dystrophix) that has the potential to fortify dystrophic muscles without side effects. Dystrophix will be injected locally, thereby affecting all muscles within a myofascial compartment.