The initial protocol was restricted to SMA type 1 patients who were not dependent on mechanical ventilation 24 h a day and for pre-symptomatic use by children with up to 3 copies of SMN2. Access to therapies for SMA type 2 patients in the Brazilian protocol only occurred in November 2022, when the national clinical protocol for SMA expanded the use of Nusinersen to include SMA type 2 patients up to 12 years of age at the start of treatment or who had preserved the ability to sit without support as well as upper limb function.25 The gene discussed is SMN2; the disease is spinal muscular atrophy, type II.