Resumo (EN)
To perform a literature review on the specific drugs used in the treatment of Spinal Muscular Atrophy (SMA), a neurodegenerative genetic disease characterized by weakness in the limbs and muscular atrophy. Narrative literature review, carried out through a descriptive study, with a qualitative approach, based on studies through a classificatory selection of research on the pharmacotherapy of SMA. The drugs approved for the treatment of AME are part of the arsenal of gene therapy: nusinersen, onasemnogeno abeparvovec and risdiplam. Except for onasemnogeno abeparvovec, used in a single dose, the others should be used by the patients for the rest of their lives. All of them, in different ways, raise the levels of the SMN (motor neuron survival) protein, whose deficiency leads to the death of motor neurons, causing the progressive symptoms of SMA. These drugs have a high cost and are difficult to obtain, are barely accessible and only nusinersen is offered by SUS. Now, pharmacological treatment alternatives are scarce and difficult to access and the cure, despite the efforts of science, is still far from reality. However, gene therapies are a differential for the treatment and control of SMA, representing an innovation and hope for patients with this disease.