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Clinical trial to test muscular dystrophy therapy developed at The Ottawa Hospital


septembre 10, 2024

Dr. Michael Rudnicki“In a paradigm shifting approach, we have developed a drug that mobilizes muscle stem cells to enhance regeneration of skeletal muscle as a treatment for this devastating lethal disease,” - Dr. Michael RudnickiResearchers at The Ottawa Hospital, the University of Ottawa and Satellos Bioscience have developed an innovative regenerative therapy for Duchenne muscular dystrophy (DMD) that is now being tested in people as part of a world-first clinical trial. DMD is an inherited disease that causes severe muscle weakness, typically resulting in an inability to walk by age 12. 

Dr. Michael Rudnicki and his team discovered that DMD affects muscle stem cells in addition to muscle fibers, compromising the body’s ability to repair ongoing muscle damage. By targeting muscle stem cells, the new small-molecule therapy is designed to help people with DMD repair and regenerate their own muscle tissue. 

Dr. Rudnicki, together with Frank Gleeson, founded Satellos Bioscience in 2018 to advance this research. The clinical trial will begin by testing the safety of the therapy in healthy volunteers, and then in people with DMD. The first phase is taking place in Australia, with subsequent phases planned for Canada and other countries. Learn more from Satellos.

“In a paradigm shifting approach, we have developed a drug that mobilizes muscle stem cells to enhance regeneration of skeletal muscle as a treatment for this devastating lethal disease,” said Dr. Michael Rudnicki, Senior Scientist and Scientific Director of Regenerative Medicine at The Ottawa Hospital, Professor at the University of Ottawa and Chief Discovery Officer at Satellos.

The Ottawa Hospital is a leading academic health, research and learning hospital proudly affiliated with the University of Ottawa. All researchers at The Ottawa Hospital follow a Responsible Innovation Framework for developing and commercializing innovations in a responsible way. 

 

Scientific Program tags: Regenerative Medicine Program