Nusinersen in type 1 spinal muscular atrophy: Twelve-month real-world data.
Marika PaneGiorgia CorattiValeria A SansoneSonia MessinaClaudio BrunoMichela CatterucciaMaria SframeliEmilio AlbamonteMarina PedemonteAdele D'AmicoChiara BravettiBeatrice BertiGiorgia BrigatiPaola TacchettiFrancesca SalminRoberto de SanctisSimona LucibelloMarco PiastraOrazio GenoveseEnrico BertiniGiuseppe VitaFrancesco Danilo TizianoEugenio Mercurinull nullPublished in: Annals of neurology (2019)
Our results, expanding the age range and the severity of type I patients treated with nusinersen over 1 year, provide additional data on the range of efficacy of the drug that will be helpful in making an informed decision on whether to start treatment in patients of different ages and severity. ANN NEUROL 2019;86:443-451.