THE STRATEGIES FOR THE TREATMENT OF SPINAL MUSCULAR ATROPHY
DOI:
https://doi.org/10.47820/recima21.v5i5.5176Keywords:
Health strategies, Therapy, Muscular Atrophy, SpinalAbstract
Spinal Muscular Atrophy (SMA) is a congenital neuromuscular disease caused by deletion or mutation in the SMN1 and SMN2 genes of chromosome 5q13, which causes progressive muscle weakness. The objective of this literature review is to understand the main strategies for the treatment of SMA, seeking to analyze the most relevant studies on the subject. This is an integrative literature review, using articles from 2017 to 2022 from the databases: PubMed and VHL. The types of therapies for the treatment of SMA addressed in the selected articles were: 58.8% splicing modulation therapy; 47% studies of new therapies; 35.3% gene-directed therapy; 5.9% neuroprotective therapy; 5.9% protein stabilization therapy and 5.9% cell replacement therapy. Based on this research, we found that SMA is still a disease without a cure. Existing pharmacological treatments, mainly Nusinersen, delay the evolution of the disease to more severe conditions, but do not recover the degeneration suffered by the motor neurons. Thus, the main objective of these therapies is to improve the quality of life of patients, increasing and preserving residual muscle function in order to prolong the life expectancy of patients.
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