New perspectives for the management of Duchenne muscular dystrophy
(2024)
Files
ThesecompleteformatlivretPDF.pdf
Open Access - Adobe PDF
- 18.56 MB
Details
- Authors
- Supervisors
- Abstract
- Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterized by chronic muscle inflammation leading to progressive muscle weakness. This condition generally results in the death of patients around the age of 30 due to cardiac or respiratory complications. Currently, corticosteroids are the only treatment used in routine clinical practice. Although they can slow the progression of the disease, they cause numerous undesirable side effects. Therefore, this work aims to discover new therapeutic approaches for treating DMD. This thesis shows that MCC950, a specific inhibitor of the inflammasome, and ALY688, an adiponectin (ApN) agonist, have proven effective in both mdx mice, the reference animal model for this disease, and muscle cells from DMD patients. These compounds have demonstrated anti-inflammatory, anti-necrotic, anti-fibrotic, and pro-myogenic effects. Finally, AdipoRon, another ApN agonist, has shown similar effects on the heart. This thesis thus highlights that the three tested medications are promising candidates for the treatment of DMD.
- Affiliations
Citations
Dubuisson, N. (2024). New perspectives for the management of Duchenne muscular dystrophy.