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. 2022 Mar;9(3):404-409.
doi: 10.1002/acn3.51514. Epub 2022 Feb 15.

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

Marika Pane  1   2 , Giorgia Coratti  1 , Maria Carmela Pera  2 , Valeria A Sansone  3 , Sonia Messina  4 , Adele d'Amico  5 , Claudio Bruno  6 , Francesca Salmin  3 , Emilio Albamonte  3 , Roberto De Sanctis  2 , Maria Sframeli  4 , Vincenzo Di Bella  4 , Simone Morando  6 , Concetta Palermo  2 , Anna Lia Frongia  1 , Laura Antonaci  1 , Anna Capasso  1 , Michela Catteruccia  5 , Antonella Longo  5 , Martina Ricci  1 , Costanza Cutrona  1 , Alice Pirola  3 , Chiara Bravetti  1 , Marina Pedemonte  6 , Noemi Brolatti  6 , Enrico Bertini  5 , Eugenio Mercuri  1 ; Italian ISMAC group
Affiliations

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

Marika Pane et al. Ann Clin Transl Neurol. 2022 Mar.

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.

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Conflict of interest statement

MCP, GC, VAS, SM, ADA, CB, FS, EA, RDS, EB, MP, and EM have been consultant for BIOGEN S.R.L. which owns patent rights to nusinersen that was used in this study.

Figures

Figure 1
Figure 1
Mean changes at 12 and 24 months subdivided by age groups. Key to figure: Panel A = HFMSE in SMA 2, Panel B = HFMSE in SMA 3, Panel C = RULM in SMA 2, Panel D = RULM in SMA 3. Light green = 12 months changes in treated patients, Dark green = 24 months changes in treated patients, Light red = 12 months changes in untreated patients, Dark red = 24 months changes in untreated patients. [Colour figure can be viewed at wileyonlinelibrary.com]
See this image and copyright information in PMC

Comment in

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