Life expectancy has improved from mid-teenage years to mid-20s with the use of glucocorticoids and beyond the third decade with ventilator support and multidisciplinary care. However, Duchenne muscular dystrophy is associated with comorbidities and is a fatal disease. Glucocorticoids prolong ambulation, but their side effects are significant. Emerging investigational therapies have surfaced over the past decade and tested in clinical trials. Gene-specific strategies include nonsense readthrough, exon skipping, gene editing, utrophin modulation, and gene replacement. Other mechanisms include muscle regeneration, antioxidants, and antifibrosis, and anti-inflammatory pathways. Potential therapies are emerging, and early diagnosis is needed to initiate treatment early enough to minimize morbidity and mortality. Newborn screening can be used to improve early diagnosis, especially for gene-specific therapeutics significantly. Read the full article here.
Have you tried any gene-specific strategies such as nonsense readthrough, exon skipping, gene editing, utrophin modulation, and gene replacement? What were the results? Share your comments and questions on this forum.
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