Nusinersen in pediatric and adult patients with type III spinal muscular atrophy

OBJECTIVE: We report longitudinal data from 144 type III SMA pediatric and adult patients treated with nusinersen as part of an international effort. METHODS: Patients were assessed using Hammersmith Functional Motor Scale Expanded (HFMSE), Revised Upper Limb Module (RULM), and 6‐Minute Walk Test (6...

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Detalles Bibliográficos
Autores principales: Pera, Maria Carmela, Coratti, Giorgia, Bovis, Francesca, Pane, Marika, Pasternak, Amy, Montes, Jacqueline, Sansone, Valeria A., Dunaway Young, Sally, Duong, Tina, Messina, Sonia, Mizzoni, Irene, D’Amico, Adele, Civitello, Matthew, Glanzman, Allan M., Bruno, Claudio, Salmin, Francesca, Morando, Simone, De Sanctis, Roberto, Sframeli, Maria, Antonaci, Laura, Frongia, Anna Lia, Rohwer, Annemarie, Scoto, Mariacristina, De Vivo, Darryl C., Darras, Basil T., Day, John, Martens, William, Patanella, Katia A., Bertini, Enrico, Muntoni, Francesco, Finkel, Richard, Mercuri, Eugenio
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8351459/
https://www.ncbi.nlm.nih.gov/pubmed/34165911
http://dx.doi.org/10.1002/acn3.51411

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