THE EFFECTIVENESS OF POSSIBLE TREATMENTS IN DUCHENNE MUSCULAR DYSTROPHY: A LITERATURE REVIEW

Abstract

Duchenne Muscular Dystrophy (DMD), a debilitating genetic condition that primarily affects male children, results from a deficiency of the protein dystrophin, essential for muscle function. Mutations in the DMD gene lead to muscle degeneration, loss of motor skills, and respiratory and heart problems. Although corticosteroids are the current standard treatment, they do not address all causes of the disease and are associated with adverse effects, leading to the search for therapeutic alternatives. The objective of this literature review was to investigate the treatments being studied for Duchenne Muscular Dystrophy and their respective effectiveness. A search for previous works was carried out on the PubMed and VHL platforms, considering the descriptors “Duchenne muscular dystrophy” and “treatment” using the Boolean operator “AND”. Articles published in the last 5 years (2019-2024) were included; articles whose studies were of the clinical trial and controlled clinical trial type and complete and free articles, and articles outside the topic covered and duplicate articles were excluded, resulting in a total of 38 scientific articles. Of the studies reviewed, 5 on Vamorolone showed that it maintained the effectiveness of corticosteroids, without their adverse effects. 4 on Viltolarsen and 2 on Golodirsen showed an increase in dystrophin expression, improving muscle function. 2 on Edasalonexent showed a reduction in muscle necrosis and inflammation. 2 on Eteplirsen demonstrated a reduction in disease progression in relation to lung function and mobility. 1 on CAP 1002, 1 on Rimeporide and 1 on Mineralocorticoid Receptor Antagonists. suggested a possible cardioprotective effect. On the other hand, 1 study on Domagrozumab and 1 on the combination of Perindopril and Bisoprolol did not find support for significant treatment effects. In conclusion, it was observed that therapies such as Vamorolone, Viltolarsen, Golodirsen, Edasalonexent, Eteplirsen, Rimeporide, CAP 1002 and Mineralocorticoid Receptor Antagonists obtained positive results in the treatment of DMD, while Domagrozumab and Perindopril and Bisoprolol were not effective. However, there is still a large gap in the treatment of DMD, making it necessary to continually search for alternative therapies for it.