Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder characterized by loss of function of SMN1 gene, and a reduced amount of the survival motor neuron (SMN) protein. The disease is categorized into five types (0-IV), with the clinical appearance ranging from progressive muscular weakness and premature death (type 0) to limited motor neuron loss and normal life expectancy (type IV). Although SMA is clinically heterogeneous, it is still the most frequent genetic cause of death in infants. Over the last years, research into treatments for spinal muscular atrophy has progressed enormously because of improved understanding of the molecular basis of SMA, disease pathogenesis, and natural history. The main 4 therapeutic strategies include SMN1 gene replacement, increased transcription of SMN2 gene into a functioning SMN protein, neuroprotection, and improvement of muscle strenght and function. The first treatment to be registered for SMA was Nusinersen (Spinraza), an antisense oligonucleotide intrathecally administered drug that modifies pre-mRNA splicing of the SMN2 gene and thus promotes increased production of functioning SMN protein. It is approved for all types of SMA, but with a better response in early treatment, which points out the need to introduce newborn genetic screening. Nusinersen is not a cure therapy, but the results of extension clinical trials are still awaited. The research result of the other potential drugs are also being awaited, that were promising so far. At the moment, the multidisciplinary clinical approach is the most important, because it affects the natural history of the disease and improves the quality of life.