Type I SMA "new natural history": long-term data in nusinersen-treated patients.
Marika PaneGiorgia CorattiValeria Ada SansoneSonia MessinaMichela CatterucciaClaudio BrunoMaria SframeliEmilio AlbamonteMarina PedemonteAdele D'AmicoChiara BravettiBeatrice BertiConcetta PalermoDaniela LeoneGiorgia BrigatiPaola TacchettiFrancesca SalminRoberto De SanctisSimona LucibelloMaria Carmela PeraMarco PiastraOrazio GenoveseEnrico Silvio BertiniGianluca VitaFrancesco Danilo TizianoEugenio Mercurinull nullPublished in: Annals of clinical and translational neurology (2021)
Our results suggest that some improvement of motor function can be observed even after the first year of treatment. This is more obvious in the infants treated in the first 2 years but some improvement can also be found in older children.