Duchenne Muscular Dystrophy Treatment
Duchenne Muscular Dystrophy is a progressive condition caused by mutations in the gene responsible for producing dystrophin, a protein essential for maintaining muscle cell stability. Without this protein, muscle fibers gradually weaken and are replaced by fibrous tissue, leading to a loss of mobility over time.
Current therapeutic strategies focus on addressing the underlying genetic defect through innovative methods such as exon-skipping. This technique allows the cell to bypass a specific mutation in the genetic code, resulting in the production of a shorter but functional version of the dystrophin protein in the body.
In addition to genetic modulation, the standard of care includes the use of corticosteroids to manage inflammation and preserve muscle strength for as long as possible. These medications help slow the rate of decline in motor and respiratory function, which is critical for extending the period during which an individual can remain independent. Newer research is exploring…