We found a match
Your institution may have access to this item. Find your institution then sign in to continue.
- Title
Nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy.
- Authors
Pane, Marika; Coratti, Giorgia; Pera, Maria Carmela; Sansone, Valeria A.; Messina, Sonia; d'Amico, Adele; Bruno, Claudio; Salmin, Francesca; Albamonte, Emilio; De Sanctis, Roberto; Sframeli, Maria; Di Bella, Vincenzo; Morando, Simone; Palermo, Concetta; Frongia, Anna Lia; Antonaci, Laura; Capasso, Anna; Catteruccia, Michela; Longo, Antonella; Ricci, Martina
- 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.
- Subjects
SPINAL muscular atrophy; NATURAL history
- Publication
Annals of Clinical & Translational Neurology, 2022, Vol 9, Issue 3, p404
- ISSN
2328-9503
- Publication type
Article
- DOI
10.1002/acn3.51514