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Lookup NU author(s): Sonia Segovia
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
BackgroundThe new treatment paradigm in Spinal Muscular Atrophy (SMA) has introduced novel phenotypes, changes in trajectories and clinical questions not fully addressed in clinical trials. To explore these challenges, several international initiatives have emerged. CuidAME was created as a nationwide clinical network in Spain designed to standardise SMA care, facilitate knowledge sharing, and capture data in a longitudinal comprehensive registry.ObjectiveEvaluating the usefulness of the CuidAME project to capture data in a real-world setting.MethodsThis multicentric cohort study includes individuals with SMA followed at participating hospitals. Clinical examinations and multidisciplinary assessments were performed during routine clinical visits. We present a cross-sectional analysis of the registry population.ResultsAs of February 2025, 543 participants from 25 hospitals were recruited: 12 were presymptomatic, 125 (23%) had SMA type 1, 208 (35%) type 2, 194 (38%) type 3, and 4 (<1%) type 4. Among the cohort, 91% (n = 495) had received at least one disease-modifier treatment, with 17 discontinuations. The registry included 5092 motor assessments, 1565 performed before treatment initiation.ConclusionsCuidAME is an academic, longitudinal, real-world data collection project that demonstrated a fast and effective model for implementation facilitating the standardization of clinical practices and outcome measures across Spain. By aligning with core dataset used in other registries and establishing multidisciplinary working groups, the initiative will contribute to sharing knowledge to advance SMA care and improve patient outcomes.
Author(s): Puig-Ram C, Segovia S, Garcia-Uzquiano R, Nungo Garzon NC, Aragon-Gawinska K, Garcia Romero M, Exposito-Escudero JM, Carrera-Garcia L, Lopez-Lobato M, Paradas C, Gonzalez Mera L, Alvarez Molinero M, Gomez Andres D, Toro E, Fernandez Ramos JA, Grimalt MA, Toledo Bravo de Laguna L, Gonzalez Barrios D, Tizzano EF, Cattinari MG, Medina J, Calvo Medina R, Munell F, Sotoca J, Martinez-Salcedo E, Moreno Escribano A, Povedano Panades M, Fernandez-Garcia MA, Pitarch-Castellano I, Vazquez-Costa JF, Natera-de Benito D, Nascimento A
Publication type: Article
Publication status: Published
Journal: Journal of Neuromuscular Diseases
Year: 2025
Volume: 12
Issue: 6
Pages: 837-848
Print publication date: 01/11/2025
Online publication date: 28/07/2025
Acceptance date: 05/07/2025
Date deposited: 27/11/2025
ISSN (print): 2214-3599
ISSN (electronic): 2214-3602
Publisher: Sage
URL: https://doi.org/10.1177/22143602251361190
DOI: 10.1177/22143602251361190
Data Access Statement: Data will be available under reasonable request.
PubMed id: 40726133
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