Annals of Clinical and Translational Neurology (Mar 2022)
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 Bertini,
- Eugenio Mercuri,
- Italian ISMAC group
Affiliations
- Marika Pane
- Pediatric Neurology Università Cattolica del Sacro Cuore Rome Italy
- Giorgia Coratti
- Pediatric Neurology Università Cattolica del Sacro Cuore Rome Italy
- Maria Carmela Pera
- Centro Clinico Nemo Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy
- Valeria A. Sansone
- The NEMO Center in Milan, Neurorehabilitation Unit University of Milan, ASST Niguarda Hospital Milan Italy
- Sonia Messina
- Department of Clinical and Experimental Medicine University of Messina Messina Italy
- Adele d'Amico
- Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders Bambino Gesù Children's Hospital, IRCCS Rome Italy
- Claudio Bruno
- Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophtalmology, Genetics, Maternal and Child Health IRCCS Istituto Giannina Gaslini and University of Genoa Genoa Italy
- Francesca Salmin
- The NEMO Center in Milan, Neurorehabilitation Unit University of Milan, ASST Niguarda Hospital Milan Italy
- Emilio Albamonte
- The NEMO Center in Milan, Neurorehabilitation Unit University of Milan, ASST Niguarda Hospital Milan Italy
- Roberto De Sanctis
- Centro Clinico Nemo Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy
- Maria Sframeli
- Department of Clinical and Experimental Medicine University of Messina Messina Italy
- Vincenzo Di Bella
- Department of Clinical and Experimental Medicine University of Messina Messina Italy
- Simone Morando
- Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophtalmology, Genetics, Maternal and Child Health IRCCS Istituto Giannina Gaslini and University of Genoa Genoa Italy
- Concetta Palermo
- Centro Clinico Nemo Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy
- Anna Lia Frongia
- Pediatric Neurology Università Cattolica del Sacro Cuore Rome Italy
- Laura Antonaci
- Pediatric Neurology Università Cattolica del Sacro Cuore Rome Italy
- Anna Capasso
- Pediatric Neurology Università Cattolica del Sacro Cuore Rome Italy
- Michela Catteruccia
- Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders Bambino Gesù Children's Hospital, IRCCS Rome Italy
- Antonella Longo
- Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders Bambino Gesù Children's Hospital, IRCCS Rome Italy
- Martina Ricci
- Pediatric Neurology Università Cattolica del Sacro Cuore Rome Italy
- Costanza Cutrona
- Pediatric Neurology Università Cattolica del Sacro Cuore Rome Italy
- Alice Pirola
- The NEMO Center in Milan, Neurorehabilitation Unit University of Milan, ASST Niguarda Hospital Milan Italy
- Chiara Bravetti
- Pediatric Neurology Università Cattolica del Sacro Cuore Rome Italy
- Marina Pedemonte
- Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophtalmology, Genetics, Maternal and Child Health IRCCS Istituto Giannina Gaslini and University of Genoa Genoa Italy
- Noemi Brolatti
- Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophtalmology, Genetics, Maternal and Child Health IRCCS Istituto Giannina Gaslini and University of Genoa Genoa Italy
- Enrico Bertini
- Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders Bambino Gesù Children's Hospital, IRCCS Rome Italy
- Eugenio Mercuri
- Pediatric Neurology Università Cattolica del Sacro Cuore Rome Italy
- Italian ISMAC group
- DOI
- https://doi.org/10.1002/acn3.51514
- Journal volume & issue
-
Vol. 9,
no. 3
pp. 404 – 409
Abstract
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.
