Nusinersen efficacy data for 24-month in type 2 and 3 spinal muscular atrophy

Marika Pane, Eugenio Maria Mercuri, Giorgia Coratti, Maria Carmela Pera, Adele D'Amico, Roberto De Sanctis, Anna Lia Frongia, Laura Antonaci, Anna Capasso, Antonella Longo, Martina Ricci, Costanza Cutrona, Enrico Silvio Bertini, Valeria A Sansone, Sonia Messina, Claudio Bruno, Francesca Salmin, Emilio Albamonte, Maria Sframeli, Vincenzo Di BellaSimone Morando, Concetta Palermo, Michela Catteruccia, Alice Pirola, Chiara Bravetti, Marina Pedemonte, Noemi Brolatti

Risultato della ricerca: Contributo in rivistaArticolo in rivistapeer review

Abstract

The study reports real world data in type 2 and 3 SMA patients treated for at least 2 years with nusinersen. Increase in motor function was observed after 12 months and during the second year. The magnitude of change was variable across age and functional subgroup, with the largest changes observed in young patients with higher function at baseline. When compared to natural history data, the difference between study cohort and untreated patients swas significant on both Hammersmith Functional Motor Scale and Revised Upper Limb Module both at 12 months and at 24 months.
Lingua originaleEnglish
pagine (da-a)404-409
Numero di pagine6
RivistaAnnals of Clinical and Translational Neurology
Volume9
DOI
Stato di pubblicazionePubblicato - 2022

Keywords

  • Nusinersen
  • spinal muscular atroophy

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