Potential biomarkers of the effectiveness of pathogenetic therapy of spinal muscular atrophy in adults

The review summarizes and systematizes data on the prognostic significance of various biomarkers in determining the effectiveness of pathogenetic therapy for spinal muscular atrophy. The review includes a clinical case of a patient with type 3 spinal muscular atrophy. The case illustrates that a decrease in the level of heavy chains of neurofilaments in the cerebrospinal fluid of the patient during the use of pathogenetic therapy with nusinersen positively correlated with an improvement in motor function, assessed by standard functional scales.