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

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

doi:10.1002/acn3.51514

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

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

Risultato della ricerca: Contributo in rivista › Articolo › peer 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 originale	Inglese
pagine (da-a)	404-409
Numero di pagine	6
Rivista	Annals of Clinical and Translational Neurology
Volume	9
DOI	https://doi.org/10.1002/acn3.51514
Stato di pubblicazione	Pubblicato - 2022

Keywords

Nusinersen
spinal muscular atroophy

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10.1002/acn3.51514

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Pane, M., Coratti, G., Pera, M. C., Sansone, V. A., Messina, S., D'Amico, A., Bruno, C., Salmin, F., Albamonte, E., De Sanctis, R., Sframeli, M., Di Bella, V., Morando, S., Palermo, C., Frongia, A. L., Antonaci, L., Capasso, A., Catteruccia, M., Longo, A., ... Mercuri, E. M. (2022). Nusinersen efficacy data for 24-month in type 2 and 3 spinal muscular atrophy. Annals of Clinical and Translational Neurology, 9, 404-409. https://doi.org/10.1002/acn3.51514

@article{4cd662f2877349438da674c6ffa0c179,

title = "Nusinersen efficacy data for 24-month in type 2 and 3 spinal muscular atrophy",

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.",

keywords = "Nusinersen, spinal muscular atroophy, Nusinersen, spinal muscular atroophy",

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

year = "2022",

doi = "10.1002/acn3.51514",

language = "English",

volume = "9",

pages = "404--409",

journal = "Annals of Clinical and Translational Neurology",

issn = "2328-9503",

publisher = "John Wiley \& Sons Inc.",

}

Pane, M , Coratti, G, Pera, MC, Sansone, VA, Messina, S, D'Amico, A, Bruno, C, Salmin, F, Albamonte, E, De Sanctis, R, Sframeli, M, Di Bella, V, Morando, S, Palermo, C, Frongia, AL, Antonaci, L, Capasso, A, Catteruccia, M, Longo, A, Ricci, M, Cutrona, C, Pirola, A, Bravetti, C, Pedemonte, M, Brolatti, N, Bertini, ES & Mercuri, EM 2022, 'Nusinersen efficacy data for 24-month in type 2 and 3 spinal muscular atrophy', Annals of Clinical and Translational Neurology, vol. 9, pagg. 404-409. https://doi.org/10.1002/acn3.51514

TY - JOUR

T1 - Nusinersen efficacy data for 24-month in type 2 and 3 spinal muscular atrophy

AU - Pane, Marika

AU - Coratti, Giorgia

AU - Pera, Maria Carmela

AU - Sansone, Valeria A.

AU - Messina, Sonia

AU - D'Amico, Adele

AU - Bruno, Claudio

AU - Salmin, Francesca

AU - Albamonte, Emilio

AU - De Sanctis, Roberto

AU - Sframeli, Maria

AU - Di Bella, Vincenzo

AU - Morando, Simone

AU - Palermo, Concetta

AU - Frongia, Anna Lia

AU - Antonaci, Laura

AU - Capasso, Anna

AU - Catteruccia, Michela

AU - Longo, Antonella

AU - Ricci, Martina

AU - Cutrona, Costanza

AU - Pirola, Alice

AU - Bravetti, Chiara

AU - Pedemonte, Marina

AU - Brolatti, Noemi

AU - Bertini, Enrico Silvio

AU - Mercuri, Eugenio Maria

PY - 2022

Y1 - 2022

N2 - 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.

AB - 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.

KW - Nusinersen

KW - spinal muscular atroophy

KW - Nusinersen

KW - spinal muscular atroophy

UR - http://hdl.handle.net/10807/197785

U2 - 10.1002/acn3.51514

DO - 10.1002/acn3.51514

M3 - Article

SN - 2328-9503

VL - 9

SP - 404

EP - 409

JO - Annals of Clinical and Translational Neurology

JF - Annals of Clinical and Translational Neurology

ER -

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

Abstract

Keywords

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