Spinal Muscular Atrophy Functional Composite Score Revised (SMA-FCR) in Untreated and Nusinersen-Treated Patient Cohorts
The SMA-FCR broadens the spectrum of abilities captured in SMA. Analyses of the treated-untreated differences in mean annual rate of change suggest that the SMA-FCR may be more sensitive to change than the HFMSE. The use of the SMA-FCR in clinical trials might allow for study designs with broader eligibility criteria including weaker individuals who score minimally on the HFMSE and stronger individuals who score maximally on the RULM.
Authors
- Adele D’Amico (ORCID: https://orcid.org/0000-0003-2438-2624)
- 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)
- Sonia Messina (ORCID: https://orcid.org/0000-0001-7994-3391)
- John Day (ORCID: https://orcid.org/0000-0002-0086-9529)
- Eugenio Maria Mercuri (ORCID: https://orcid.org/0000-0002-9851-5365)
- Zarazuela Zolkipli‐Cunningham (ORCID: https://orcid.org/0000-0002-0743-8879)
- Darryl C. De Vivo (ORCID: https://orcid.org/0000-0001-8420-0795)
- Basil T. Darras (ORCID: https://orcid.org/0000-0002-1536-8145)
- William B. Martens (ORCID: https://orcid.org/0009-0006-6261-3701)
- Michael P. McDermott (ORCID: https://orcid.org/0000-0001-7443-9145)
- Claudio Bruno (ORCID: https://orcid.org/0000-0002-3426-2901)
- Amy Pasternak
Institutions
- University of Messina (IT)
- Boston Children's 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)
- Istituti di Ricovero e Cura a Carattere Scientifico (IT)
- University of Rochester (US)
- Philadelphia University (US)
- Stanford University (US)
Publication Details
- Journal
- Neurology
- Published
- 2025-06-27
- DOI
- https://doi.org/10.1212/wnl.0000000000213839
- Citations
- 1
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 1.63