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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...

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Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2021
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.
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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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