{
  "abstract": "The 2018 international SMA care guidelines were developed before disease-modifying treatments transformed SMA progression and prognosis. Focused on paediatric care, they were based on minimal standards and did not address the evolving phenotypes resulting from new treatments.SMA CARE UK, a collaborative initiative involving patients, healthcare professionals and stakeholders, was created to update, harmonise and support the implementation of evidence-based care standards for SMA across the UK. The project is hosted by SMA UK and rolled out over 3 years with the support of the University of Newcastle and the SMA Reach clinical networks. Expert working groups, including patient representatives and clinical specialists, focus on specific care topics.The current guidelines are being reviewed to identify areas needing revision based on new evidence, changing practices and unmet needs. It aims to create relevant, evidence-based recommendations for specific care areas, highlight knowledge gaps and explore strategies to gather necessary evidence.Final recommendations will be published in professional and patient-friendly formats after consultation with clinical and patient networks. Endorsement will be sought from professional bodies and findings will be presented at conferences.The initiative also aims to establish a UK minimum standard of care to address regional inequalities, support families and promote dialogue about optimal management. Collaboration with global networks will enhance research, data collection, and alignment with international standards.vanessa.christie-brown@smauk.org.uk",
  "authors": [
    {
      "affiliations": [
        "Leeds Teaching Hospitals NHS Trust, Leeds, UK"
      ],
      "name": "Childs Anne-Marie"
    },
    {
      "affiliations": [
        "Spinal Muscular Atrophy UK",
        "The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute"
      ],
      "name": "Christie-Brown Vanessa"
    },
    {
      "affiliations": [
        "The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute"
      ],
      "name": "Alvarez Grecia"
    },
    {
      "affiliations": [
        "The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK"
      ],
      "name": "Baranello Giovanni"
    },
    {
      "affiliations": [
        "The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK"
      ],
      "name": "Fitzpatrick Claire"
    },
    {
      "affiliations": [
        "Department of Paediatric Neurology, Royal Hospital for Children, Glasgow, UK"
      ],
      "name": "Horrocks Iain"
    },
    {
      "affiliations": [
        "Manchester Centre for Clinical Neurosciences, Salford Royal NHS Foundation Trust, Salford, UK"
      ],
      "name": "Lilleker James"
    },
    {
      "affiliations": [
        "The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute"
      ],
      "name": "Muni-Lofra Robert"
    },
    {
      "affiliations": [
        "The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK"
      ],
      "name": "Scoto Mariacristina"
    },
    {
      "affiliations": [
        "The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute"
      ],
      "name": "Marini-Bettolo Chiara"
    }
  ],
  "title": "45 SMA care UK: ensuring the best care for individuals with SMA across the UK",
  "uid": "4642a6e4-f247-5ba5-945f-4a0e3bcb7634"
}