Nusinersen safety and effects on motor function in adult spinal muscular atrophy type 2 and 3.
Lorenzo MaggiLuca BelloSilvia BonannoAlessandra GovoniClaudia CaponnettoLuigia PassamanoMarina GrandisFrancesca TrojsiFederica CerriManfredi FerraroVirginia BozzoniLuca CaumoRachele PirasRaffaella TanelElena SaccaniMegi MeneriVeria VacchianoGiulia RicciGianni SorarùEustachio D'ErricoIrene TramacereSara BortolaniGiovanni PavesiRiccardo ZaninMauro SilvestriniLuisa PolitanoAngelo SchenonePrevitali Stefano CarloAngela BerardinelliMara TurriLorenzo VerrielloMichela CocciaRenato MantegazzaRocco LiguoriMassimiliano MirabellaGianni MarrosuGabriele SicilianoIsabella Laura SimoneTiziana MonginiGiacomo ComiElena PegoraroPublished in: Journal of neurology, neurosurgery, and psychiatry (2020)
Our data provide further evidence of nusinersen safety and efficacy in adult SMA2 and SMA3, with the latter appearing to be cumulative over time. In patients with extremely advanced disease, effects on residual motor function are less clear.