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Lead investigator

Collaborators & Co-Investigators

• Alexander Green, PhD (Co-PI)
• Junio Dort, PhD
• Vincent Mouly, PhD
• Michael De Lisio, PhD

Research Sites & Affiliations

• University of Ottawa, Ottawa, Ontario

Budget: $99,906.54

Disorders: All Neuromuscular Disorders (cross-cutting) ,   Duchenne/Becker Muscular Dystrophy

Research Areas: Discover Novel Treatments & Therapies

Abstract: 

Duchenne Muscular Dystrophy (DMD) is a serious disease that mainly affects boys, with about 1 in every 3,500 males being affected. Muscles from people with DMD get damaged when they contract, leading to muscle weakness. Unlike muscle from healthy individuals, the muscles can’t repair themselves properly, and this gets worse over time. Right now, there is no cure for DMD, and even the gene and cell therapies in development might not completely fix the problem. The most used treatment is a type of medication called corticosteroids. They can help people with DMD live longer, but using them for a long time can cause problems like muscle shrinking, metabolism issues, and altered hormone levels. This study aims to find other drugs that can fix muscle cell damage without causing problems like corticosteroids do. Based on current research, it is possible that existing medications might be able to reduce the damage to muscle cell membranes when they contract in DMD. To test this idea, this study aims to develop a new way to examine muscle damage within the lab. This method can be used to test medications that are already approved for other purposes to see if they can reduce muscle damage and help with repair. Through this research, new ways to use existing medicines can be developed that are safe for people with DMD.

Impact: