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2. Nusinersen safety and effects on motor function in adult spinal muscular atrophy type 2 and 3

Author

Veria Vacchiano, Luca Bello, Renato Mantegazza, Eustachio D'Errico, Tiziana Mongini, Riccardo Zanin, Gabriele Siciliano, Francesca Trojsi, Elena Saccani, Federica Cerri, Marina Grandis, Michela Coccia, Stefano C. Previtali, Alessandra Govoni, Massimiliano Filosto, L. Passamano, Giovanni Pavesi, Luisa Politano, Mauro Silvestrini, Claudia Caponnetto, Silvia Bonanno, Virginia Bozzoni, Manfredi Ferraro, Elena Pegoraro, Raffaella Tanel, Lorenzo Verriello, Angelo Schenone, Sara Bortolani, Lorenzo Maggi, Angela Berardinelli, Giacomo P. Comi, Megi Meneri, Rocco Liguori, Gianni Sorarù, G. Marrosu, Mara Turri, Luca Caumo, Irene Tramacere, Rachele Piras, Giulia Ricci, Isabella Laura Simone, Maggi, Lorenzo, Bello, Luca, Bonanno, Silvia, Govoni, Alessandra, Caponnetto, Claudia, Passamano, Luigia, Grandis, Marina, Trojsi, Francesca, Cerri, Federica, Ferraro, Manfredi, Bozzoni, Virginia, Caumo, Luca, Piras, Rachele, Tanel, Raffaella, Saccani, Elena, Meneri, Megi, Vacchiano, Veria, Ricci, Giulia, Soraru', Gianni, D'Errico, Eustachio, Tramacere, Irene, Bortolani, Sara, Pavesi, Giovanni, Zanin, Riccardo, Silvestrini, Mauro, Politano, Luisa, Schenone, Angelo, Previtali, Stefano Carlo, Berardinelli, Angela, Turri, Mara, Verriello, Lorenzo, Coccia, Michela, Mantegazza, Renato, Liguori, Rocco, Filosto, Massimiliano, Marrosu, Gianni, Siciliano, Gabriele, Simone, Isabella Laura, Mongini, Tiziana, Comi, Giacomo, Pegoraro, Elena, Maggi L., Bello L., Bonanno S., Govoni A., Caponnetto C., Passamano L., Grandis M., Trojsi F., Cerri F., Ferraro M., Bozzoni V., Caumo L., Piras R., Tanel R., Saccani E., Meneri M., Vacchiano V., Ricci G., Soraru' G., D'Errico E., Tramacere I., Bortolani S., Pavesi G., Zanin R., Silvestrini M., Politano L., Schenone A., Previtali S.C., Berardinelli A., Turri M., Verriello L., Coccia M., Mantegazza R., Liguori R., Filosto M., Marrosu G., Siciliano G., Simone I.L., Mongini T., Comi G., and Pegoraro E.

Subjects
Male, Pediatrics, spinal muscular atrophy type 3, Vital Capacity, spinal muscular atrophy type 2, Oligonucleotides, Walking, Spinal Muscular Atrophies of Childhood, Cohort Studies, Efficacy, 0302 clinical medicine, Forced Expiratory Volume, Age of Onset, Young adult, Injections, Spinal, Sitting Position, 0303 health sciences, Middle Aged, Psychiatry and Mental health, Treatment Outcome, Italy, Female, Nusinersen, Cohort study, Adult, medicine.medical_specialty, Adolescent, Spinal, Walk Test, Injections, Young Adult, 03 medical and health sciences, Rating scale, medicine, Humans, Aged, Functional Status, Oligonucleotides, Antisense, Retrospective Studies, Antisense, 030304 developmental biology, business.industry, Retrospective cohort study, Spinal muscular atrophy, medicine.disease, Surgery, Neurology (clinical), Age of onset, business, 030217 neurology & neurosurgery
Abstract

ObjectiveTo retrospectively investigate safety and efficacy of nusinersen in a large cohort of adult Italian patients with spinal muscular atrophy (SMA).MethodsInclusion criteria were: (1) clinical and molecular diagnosis of SMA2 or SMA3; (2) nusinersen treatment started in adult age (>18 years); (3) clinical data available at least at baseline (T0-beginning of treatment) and 6 months (T6).ResultsWe included 116 patients (13 SMA2 and 103 SMA3) with median age at first administration of 34 years (range 18–72). The Hammersmith Functional Rating Scale Expanded (HFMSE) in patients with SMA3 increased significantly from baseline to T6 (median change +1 point, pConclusionsOur data provide further evidence of nusinersen safety and efficacy in adult SMA2 and SMA3, with the latter appearing to be cumulative over time. In patients with extremely advanced disease, effects on residual motor function are less clear.

Published
2020

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