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Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era
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 managem...
Autores principales: | , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
IOS Press
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8385518/ https://www.ncbi.nlm.nih.gov/pubmed/33646175 http://dx.doi.org/10.3233/JND-200611 |
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author | Walter, Maggie C. Chiriboga, Claudia Duong, Tina Goemans, Nathalie Mayhew, Anna Ouillade, Laëtitia Oskoui, Maryam Quinlivan, Ros Vázquez-Costa, Juan F. Vissing, John Servais, Laurent |
author_facet | Walter, Maggie C. Chiriboga, Claudia Duong, Tina Goemans, Nathalie Mayhew, Anna Ouillade, Laëtitia Oskoui, Maryam Quinlivan, Ros Vázquez-Costa, Juan F. Vissing, John Servais, Laurent |
author_sort | Walter, Maggie C. |
collection | PubMed |
description | 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 action 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. |
format | Online Article Text |
id | pubmed-8385518 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | IOS Press |
record_format | MEDLINE/PubMed |
spelling | pubmed-83855182021-09-09 Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era Walter, Maggie C. Chiriboga, Claudia Duong, Tina Goemans, Nathalie Mayhew, Anna Ouillade, Laëtitia Oskoui, Maryam Quinlivan, Ros Vázquez-Costa, Juan F. Vissing, John Servais, Laurent J Neuromuscul Dis Short Communication 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 action 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. IOS Press 2021-07-30 /pmc/articles/PMC8385518/ /pubmed/33646175 http://dx.doi.org/10.3233/JND-200611 Text en © 2021 – The authors. Published by IOS Press https://creativecommons.org/licenses/by-nc/4.0/This is an open access article distributed under the terms of the Creative Commons Attribution Non-Commercial (CC BY-NC 4.0) License (https://creativecommons.org/licenses/by-nc/4.0/) , which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited. |
spellingShingle | Short Communication Walter, Maggie C. Chiriboga, Claudia Duong, Tina Goemans, Nathalie Mayhew, Anna Ouillade, Laëtitia Oskoui, Maryam Quinlivan, Ros Vázquez-Costa, Juan F. Vissing, John Servais, Laurent Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era |
title | Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era |
title_full | Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era |
title_fullStr | Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era |
title_full_unstemmed | Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era |
title_short | Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era |
title_sort | improving care and empowering adults living with sma: a call to action in the new treatment era |
topic | Short Communication |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8385518/ https://www.ncbi.nlm.nih.gov/pubmed/33646175 http://dx.doi.org/10.3233/JND-200611 |
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