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

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  • Maggie C. Walter
  • Claudia Chiriboga
  • Tina Duong
  • Nathalie Goemans
  • Anna Mayhew
  • Laëtitia Ouillade
  • Maryam Oskoui
  • Ros Quinlivan
  • Juan F. Vázquez-Costa
  • Vissing, John
  • Laurent Servais

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.

Original languageEnglish
JournalJournal of Neuromuscular Diseases
Volume8
Issue number4
Pages (from-to)543-551
Number of pages9
ISSN2214-3599
DOIs
Publication statusPublished - 2021

Bibliographical note

Publisher Copyright:
© 2021 - The authors. Published by IOS Press.

    Research areas

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

ID: 301348635