Longitudinal Assessment of 4‐Year <scp>HFMSE</scp> Changes in <scp>SMA II</scp> and <scp>III</scp> Patients Treated With Nusinersen
Our results confirm that, given a follow up of 4 years, there is a persistent impact of nusinersen on clinical progression that is better observed in younger patients with higher HFMSE scores at baseline, especially during the first 2 years of treatment.
Authors
- Adele D’Amico (ORCID: https://orcid.org/0000-0003-2438-2624)
- Francesca Bovis (ORCID: https://orcid.org/0000-0002-3823-4000)
- Valeria Sansone (ORCID: https://orcid.org/0000-0002-1354-3800)
- Jacqueline Montes (ORCID: https://orcid.org/0000-0002-7357-0819)
- Tina Duong (ORCID: https://orcid.org/0000-0003-3177-4722)
- Giorgia Coratti (ORCID: https://orcid.org/0000-0001-6666-5628)
- Allan M. Glanzman (ORCID: https://orcid.org/0000-0002-7145-2608)
- Enrica Rolle
- Sally Dunaway Young (ORCID: https://orcid.org/0000-0001-9322-0608)
- Federica Ricci (ORCID: https://orcid.org/0000-0003-2019-612X)
- Ilaria Cavallina
- Simone Morando
- G Gadaleta (ORCID: https://orcid.org/0000-0001-6563-8222)
- Sonia Messina (ORCID: https://orcid.org/0000-0001-7994-3391)
- Tiziana Mongini
- John Day (ORCID: https://orcid.org/0000-0002-0086-9529)
- Eugenio Maria Mercuri (ORCID: https://orcid.org/0000-0002-9851-5365)
- Maria Carmela Pera (ORCID: https://orcid.org/0000-0001-6777-1721)
- Zarazuela Zolkipli‐Cunningham (ORCID: https://orcid.org/0000-0002-0743-8879)
- Chiara Bravetti
- Marika Pane (ORCID: https://orcid.org/0000-0002-4851-6124)
- Irene Mizzoni
- Basil T. Darras (ORCID: https://orcid.org/0000-0002-1536-8145)
- Emilio Albamonte (ORCID: https://orcid.org/0000-0002-8465-4460)
- Richard S. Finkel (ORCID: https://orcid.org/0000-0002-9351-7054)
- Michela Catteruccia (ORCID: https://orcid.org/0000-0003-0785-5004)
- Noemi Brolatti
- Matthew Civitello (ORCID: https://orcid.org/0000-0002-7858-9447)
- Roberto De Sanctis
- Michio Hirano (ORCID: https://orcid.org/0000-0002-7070-7402)
- Maria Sframeli
- Claudio Bruno
- Enrico Bertini
- Amy Pasternak
Institutions
- Università Cattolica del Sacro Cuore (IT)
- University of Messina (IT)
- University of Parma (IT)
- Boston Children's Hospital (US)
- St. Jude Children's Research Hospital (US)
- Children's Hospital of Philadelphia (US)
- Harvard University (US)
- University of Milan (IT)
- Columbia University Irving Medical Center (US)
- Istituto Giannina Gaslini (IT)
- Agostino Gemelli University Polyclinic (IT)
- Centro Clinico Nemo (IT)
- Bambino Gesù Children's Hospital (IT)
- University of Turin (IT)
- University of Pennsylvania (US)
- University of Genoa (IT)
- Stanford University (US)
Publication Details
- Journal
- European Journal of Neurology
- Published
- 2025-06-27
- DOI
- https://doi.org/10.1111/ene.70268
- Citations
- 4
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 6.50
Funders
- Novartis
- Roche
- Biogen
- Ministero della Salute