Nusinersen in spinal muscular atrophy type 1 patients older than 7 months: A cohort study

Aragon-Gawinska K, Seferian AM, Daron A, Gargaun E, Vuillerot C, Cances C, Ropars J, Chouchane M, Cuppen I, Hughes I, Illingworth M, Marini-Bettolo C, Rambaud J, Taytard J, Annoussamy M, Scoto M, Gidaro T, Servais L.
Neurology. 2018. 91(14), e1312-e1318.
The study evaluate the safety and clinical efficacy of Nusinersen in patients with Spinal Muscular Atrophy Type 1 (SMA1) older than 7 months.
Motor function was assessed with the modified Hammersmith Infant Neurologic Examination Part 2 (HINE-2) and scales adjusted to patient age, such as MFM-20 or MFM-32.

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