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Lead investigator

Collaborators & Co-Investigators

• Thomas Hawke, PhD
• Mark Tarnopolsky, MD, PhD

Research Sites & Affiliations

• York University , Toronto, Ontario
• McMaster University, Hamilton, Ontario

Budget: $100,000

Disorders: Duchenne/Becker Muscular Dystrophy

Research Areas: Discover Novel Treatments & Therapies

Abstract: 

Steroid drugs and creatine are used for muscle weakness in people living with Duchenne muscular dystrophy. Steroid drugs can also cause problems with weakening mitochondria, which are the energy producing parts of muscle cells. Duchenne also makes it harder for mitochondria to use creatine for energy. This means that both treatments may not be doing the best job possible if they are also weakening mitochondria.

We discovered that two specific drugs can offset the poor metabolism that may prevent steroid drugs and creatine from helping people as much as possible. This means that these new drugs might help the other drugs work better in people with Duchenne. This study will determine if these new drugs help muscle cells from people with Duchenne produce more energy. We will also test these drugs in mice with this disorder.

We believe the results of this study will help create new drugs that will improve current treatments. If true, this could mean that people with Duchenne will have a better quality of life.

Impact: