Nusinersen is the only treatment available, as a disease modifier, for spinal muscular atrophy (SMA). Its use and experience is limited in Argentina to know under the regime of compassionate use. To evaluate response to intrathecal nusinersen in a group of patients. Nusinersen was started in 11 patients. Of these, 7 completed induction (range 2-18 year old, 5 boys, one with SMA 1, six with SMA and reached an evaluation point of 6 months from the start of treatment. In this group, WHO milestones, Hammersmith, ULM, respiratory parameters, tolerance to treatments were evaluated.

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