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Lead investigator

Collaborators & Co-Investigators

• Aymeric Ravel-Chapuis, PhD (PI)
• Élise Duchesne, PhD (PI)

Research Sites & Affiliations

• University of Ottawa, Ottawa, Ontario
• Université du Québec à Chicoutimi, Chicoutimi, Québec

Budget: $100,000

Disorders: Myotonic Dystrophy

Research Areas: Understand Diagnosis and Disorder Progression ,   Discover Novel Treatments & Therapies

Abstract: 

Myotonic Dystrophy type 1 (DM1) is a disorder affecting many organs of the body. There is currently no cure or effective treatment for the disorder. In DM1, muscles are weaker, painful and have difficulties to relax. On the molecular level multiple signaling pathways have been reported to be altered including the AMPK signaling, which is important for energy in cells. In our recent work, we found that in cell models of DM1, AMPK signaling is repressed and when it is stimulated, it improves the pathology of these cells. AMPK can be stimulated pharmacologically and physiological (i.e. through exercise.) However, it remains unknown if there is any therapeutic benefit for persons living with DM1.

In this study, we therefore propose to take our findings from cell models to further investigate the role of AMPK in persons living with DM1 and test whether endurance training has the same beneficial effects on AMPK signaling. In addition, while exercise has been shown to be beneficial for DM1 mouse models and for DM1 patients, the impact of training programs on the disorder has not been assessed.
This study will help us better understanding myotonic dystrophy, the role of exercise in muscle health and shed light on potential targets that can be developed as novel therapies for DM1.

Impact: