Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era.

Fecha de publicación: 01/01/2021 Fecha Ahead of Print: 24/02/2021

Autores de IIS La Fe

Juan Francisco Vázquez Costa

Autor

Participantes ajenos a IIS La Fe

Walter MC
Chiriboga C
Duong T
Goemans N
Mayhew A
Ouillade L
Oskoui M
Quinlivan R
Vissing J
Servais L

Grupos

Patología Neuromuscular y Ataxias

Abstract

While Spinal Muscular Atrophy (SMA) has historically been managed with supportive measures, the emergence of innovative medicines has given those living with SMA hope for improved quality of life and has revolutionized care. Despite these advances, the use of therapies and changes in disease management strategies have focused on pediatric populations, leaving adults living with SMA, and those transitioning into adulthood, relatively neglected. Through a multi-faceted approach that gathered unbiased perspectives from clinical experts, validated insights from individuals with lived experiences, and substantiated findings with evidence from the literature, we have exposed unmet needs that are hindering the field and, ultimately, impacting care and quality of life for adults living with SMA. Here, we set new aspirations and calls to actions to inspire continued research in this field, stimulate dialogue across the SMA community and inform policies that deliver effective management and care throughout an adult's journey living with SMA.

Datos de la publicación

ISSN/ISSNe:: 2214-3599, 2214-3602
Tipo:: Article
Páginas:: 543-551
DOI:: 10.3233/JND-200611
PubMed:: 33646175
Factor de Impacto:: 1,054 SCImago ℠
Cuartil:: Q1 SCImago ℠

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Métricas

Filiaciones

Walter MC:: Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich, Bavaria, Germany
Chiriboga C:: Department of Neurology, Columbia University Irving Medical Center, New York, NY, USA
Duong T:: Stanford University, Stanford, CA, United States
Goemans N:: Faculty of Medicine, University of Leuven, University Hospitals Leuven, Leuven, Flemish Brabant, Belgium
Mayhew A:: Newcastle University Translational and Clinical Research Institute, Newcastle upon Tyne, UK
Oskoui M:: Departments of Pediatrics and Neurology, McGill University, Montreal, Quebec, Canada
Quinlivan R:: MRC Centre for Neuromuscular Diseases, UCL Institute of Neurology, National Hospital for Neurology and Neurosurgery, London, UK
Vázquez-Costa JF:: Motor neuron disease Unit, Department of Neurology, Hospital Universitario y Politécnico La Fe, Valencia, Valencia, Spain
Vissing J:: Copenhagen Neuromuscular Center, Rigshospitalet, University of Copenhagen, Copenhagen, Copenhagen, Denmark
Servais L:: MDUK Neuromuscular Center, Department of Paediatrics, University of Oxford, Oxford, UK

Keywords

Adults living with SMA, Burden of Disease (BoD), Neuromuscular Disease (NMD), Spinal Muscular Atrophy (SMA), health services, lived experience, quality of life (QoL), transition

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