Long‐term natural history in type <scp>II</scp> and <scp>III</scp> spinal muscular atrophy: a 4‐year international study on the Hammersmith Functional Motor Scale Expanded
Our findings provide natural history reference data for comparison with long-term follow-up of clinical trials or real-world data, highlighting the need to define patterns of changes in smaller SMA subgroups instead of reporting mean changes across an entire SMA cohort.
Authors
- Adele D’Amico (ORCID: https://orcid.org/0000-0003-2438-2624)
- Giovanni Baranello (ORCID: https://orcid.org/0000-0003-4871-6692)
- 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)
- Sally Dunaway Young (ORCID: https://orcid.org/0000-0001-9322-0608)
- Mariacristina Scoto (ORCID: https://orcid.org/0000-0002-7320-6443)
- Simone Morando
- Andres Nascimiento Osorio
- J. Expósito (ORCID: https://orcid.org/0000-0002-6233-823X)
- Sonia Messina (ORCID: https://orcid.org/0000-0001-7994-3391)
- Liesbeth De Waele (ORCID: https://orcid.org/0000-0001-7126-575X)
- N. Goemans
- John Day (ORCID: https://orcid.org/0000-0002-0086-9529)
- Eugenio Maria Mercuri (ORCID: https://orcid.org/0000-0002-9851-5365)
- Evelin Milev (ORCID: https://orcid.org/0000-0002-7129-1824)
- Maria Carmela Pera (ORCID: https://orcid.org/0000-0001-6777-1721)
- Zarazuela Zolkipli‐Cunningham (ORCID: https://orcid.org/0000-0002-0743-8879)
- Marika Pane (ORCID: https://orcid.org/0000-0002-4851-6124)
- Annemarie Rohwer (ORCID: https://orcid.org/0000-0002-9072-8235)
- Francesco Muntoni
- Irene Mizzoni
- Basil T. Darras (ORCID: https://orcid.org/0000-0002-1536-8145)
- Francesca Salmin
- 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)
- Boston Children's Hospital (US)
- Hospital Sant Joan de Déu Barcelona (ES)
- 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)
- Great Ormond Street Hospital (GB)
- Great Ormond Street Hospital for Children NHS Foundation Trust (GB)
- Instituto de Salud Carlos III (ES)
- Istituto Giannina Gaslini (IT)
- Agostino Gemelli University Polyclinic (IT)
- Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda (IT)
- Centre for Biomedical Network Research on Rare Diseases (ES)
- Centro Clinico Nemo (IT)
- Bambino Gesù Children's Hospital (IT)
- Institut de Recerca Sant Joan de Déu
- University College London (GB)
- University of Pennsylvania (US)
- University of Genoa (IT)
- Philadelphia University (US)
- Stanford University (US)
- KU Leuven (BE)
Publication Details
- Journal
- European Journal of Neurology
- Published
- 2024-10-11
- DOI
- https://doi.org/10.1111/ene.16517
- Citations
- 8
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 3.50
Funders
- Novartis
- Biogen
- Roche Italia
- Ministero della Salute