Safety, tolerability and clinical efficacy of nusinersen in SMA type 1 older than 7 months: a prospective study

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The safety and efficacy of intrathecal nusinersen has been demonstrated in SMA type 1 patients younger than 7 months and in 2–12 years old SMA type II patients. However, we lack safety and efficacy data in SMA type 1 patients older than 7 months. We present a prospective observational study of 26 patients with spinal muscular atrophy type I treated with nusinersen in Paris, all aged more than 7 months at the start of the therapy. The treatment was administered by the schedule – day 1, day 15, day 29, day 64, day 184, intrathecally, with local anesthesia.

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