[Modern principles of therapy for patients with spinal muscular atrophy].

Abstract

Spinal muscular atrophy (SMA) is a common cause of childhood mortality among hereditary diseases of the central nervous system, which are caused by the processes of degeneration and death of motor neurons of the anterior horns of the spinal cord. An urgent issue of modern neurology is pathogenetic therapy for this group of patients, the purpose of which is to increase the level of motoneuron survival protein. We performed a search on current methods of treating SMA in Web of Science, Scopus, PubMed, Embase by the keywords: spinal muscular atrophy, neuromuscular diseases, pathogenetic therapy. Significant progress has been made in the treatment of SMA over the past 7 years. A major advance is the introduction of disease-modifying therapies using SMN2 splicing modulation or gene replacement therapy. At the moment, there are 3 FDA-approved drugs for pathogenetic therapy: Nusinersen, Risdiplam, Zolgensma. The article compares the drugs, evaluates their safety and effectiveness according to the available literature. Modern drugs for the pathogenetic therapy of SMA are highly effective and reduce the mortality rate. The results of clinical trials predict the emergence of new modern drugs. This suggests a favorable prognosis for the treatment of patients with SMA.