EFFICACY, SAFETY AND ADHERENCE OF NUSINERSEN IN THE TREATMENT OF SPINAL MUSCULAR ATROPHY: AN INTEGRATIVE REVIEW OF THE LITERATURE

Abstract

Spinal Muscular Atrophy (SMA) is an autosomal recessive hereditary neurodegenerative disease, which can be subdivided into five types. It causes severe muscle weakness and atrophy of skeletal muscles, having a very negative impact on its sufferers. The objective of this review was to analyze the use of the drug nusinersen for the treatment of SMA, through its effectiveness, adverse effects and adherence among patients. A search for previous works was carried out on the PubMed and VHL platforms, and a total of 26 scientific articles were included after using the inclusion and exclusion criteria. Through the studies analyzed, it was observed that nusinersen has a very positive impact on the evolution of the disease, causing a clinical improvement in motor and respiratory function, or a stabilization of the evolution. Adverse effects were minimal, mostly mild and related to the lumbar puncture. Adherence to the medication was low due to the complexity of the treatment.  In conclusion, it is important to encourage the use of medication, as it modifies the natural history of the disease, positively impacting the lives of these patients, reducing complications and unfavorable progression of the disease.