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Duchenne muscular dystrophy (DMD) is a serious genetic disorder characterized by progressive muscle weakness and degeneration due to abnormalities in the protein dystrophin, which helps maintain muscle cells
DMD symptoms usually occur in early infancy, with a gradual loss of muscle function as the patient matures
"Muscular dystrophy is a devastating disease that affects children from a very early age
Existing cancer treatments could be used to treat muscular dystrophy, the University of British Columbia (UBC) School of Biomedical Engineering has revealed
Dr Farshad Babaeijandaghi, a postdoc at UBC and lead author of the study, said: "This class of drug has already been used in clinical trials to treat rare cancers
The finding surprised researchers who originally studied the role of macrophages, a type of white blood cell, in muscle regeneration
In experiments in mice, they found that CSF1R inhibitors (which deplete resident macrophages) had the unexpected effect of making muscle fibers more resistant to the tissue damage caused by contraction, a hallmark of muscular dystrophy
After making this discovery, the researchers tested the drug on mice with DMD
The researchers say further studies are needed to determine whether CSF1R is effective in treating DMD in humans
"Developing a new drug can be a long process," said Dr.
Reference: Metabolic reprogramming of skeletal muscle by resident macrophages points to CSF1R inhibitors as muscular dystrophy therapeutics
