The discovery of the genetic background and pathophysiology of congenital spinal muscular atrophy has enabled the development of drugs that have radically changed the course of the disease and the quality of life of patients. Since the best results are achieved when therapy is initiated at an early, asymptomatic stage, and this is only possible by screening for SMA in newborns, the international approach is to introduce universal screening. This publication describes the currently available treatment options, the importance ol neonatal SMA screening, and relevant experiences in the international setting and in Hungary.  Kulcsszavak: quatity of tife, genetic-based SMN-dependent disease-modifying therapies, spinal muscular atrophy, neonatal SMA screening