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

Maggie C Walter, Claudia Chiriboga, Tina Duong, Nathalie Goemans, Anna Mayhew, Laëtitia Ouillade, Maryam Oskoui, Ros Quinlivan, Juan F Vázquez-Costa, John Vissing, 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
Issue number4
Pages (from-to)543-551
Number of pages9
Publication statusPublished - 2021


  • Cost of Illness
  • Humans
  • Muscular Atrophy, Spinal/therapy
  • Patient Participation
  • Quality Improvement
  • Quality of Life


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