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For Patients and Families

For Patients and Families

There are many opportunities to bring your experiences and expert perspectives to specific areas of the SMA Care project. If you would like to join an expert working group, or review the draft guidelines as they are produced, then please register your interest here.

Alternatively, if you have experience in any of the proposed care areas and would like to inform a working group, then please contact the Project Manager, vanessa.christie-brown@smauk.org.uk

You can find links to a number of open surveys, studies, consultations and campaigns. The surveys and studies on this page focus on the care and management of SMA.

Would you like to share your perspective on particular areas of care? Please find here the links to live studies:

Transition: to access this survey, please click here

Exercise: there are currently 2 studies related to exercise in SMA:

  1. Exercise Behaviour Study (views and expectations around exercise): researchers at the University of Newcastle are looking at understanding the factors that either enable or restrict activity levels amongst people with neuromusuclar conditions. This will allow them to find better ways to promote healthy lifestyles. To help them, please complete this survey. It should take about 10 minutes of your time.
  2. Virtual Rehabilitation in SMA (VRehabSMA): researchers at the University of Oxford are recruiting children, young people and families living with SMA to help develop innovative virtual reality (VR)-based rehabilitation tools.The study includes:
    • A 20 minute online survey to explore exercise, gaming, technology use and views on virtual reality-based rehabilitation. It closes in September 2026 and is for:
      * young people aged 12–18 years living with SMA,
      * parents and caregivers of children and young people aged 6–18 years living with SMA
    • A device-testing study (Oxford University): children and young people aged 6–18 years with SMA are invited to test an early prototype VR rehabilitation system. The research team is particularly keen to reruit additional participants and welcomes interest from families outside the Oxford area.Participation involves:
      • A pre-visit assessment
      • Two visits to the University of Oxford
      • Feedback questionnaires and safety follow-up

Further Information

Your participation is hugely appreciated.

Please note: SMA Care UK is sharing these research opportunities on behalf of the study teams. Inclusion on this page does not constitute endorsement of the study by SMA Care UK.

  • Essential Information for Emergency Services When Assessing and Caring for a Child with Spinal Muscular Atrophy (2023)

    Every child with spinal muscular atrophy (SMA) should have an individual Emergency Healthcare Plan (EHP). This written plan outlines key actions and information for medical teams to follow if the child becomes unwell.In some situations, families may need to take their child to Accident & Emergency (A&E) services, often at hospitals where staff may be unfamiliar with SMA. To support these teams, we’ve worked with clinical experts to develop a helpful information sheet for families to share alongside the child’s EHP. This resource provides essential guidance to help A&E staff assess and respond appropriately to the needs of a child with SMA. You can access the document here.
  • Adults’ Perspectives ReportThis report focuses on the experiences, priorities and recommendations of adults living with SMA in the UK. These were obtained through a couple of workshops held in January 2025 in Manchester and February 2025 in London. You can read the report here.
  • The Guide to the 2017 International Standards of Care for SMA

    Clinicians, academics, health care professionals and patient representatives from Spinal Muscular Atrophy UK, Cure SMA, SMA Europe, Muscular Dystrophy UK and TREAT-NMD have used the 2017 care recommendations, to work together to produce a current and comprehensive medical guide aimed at people with SMA and their families. The guide can be accessed here.

  • The Big Weekend – Nottingham University – 27th September 2025Listen to Dr. Anne-Marie Childs, Lisa Edel, Professor Giovanni Baranello and Dr. Robert Muni-Lofra discuss the importance of multidisciplinary care for different patient groups, across respiratory care, and spines and hips management.They introduce SMA Care UK, a collaborative initiative to update the standards of care and promote best practice, from the first stages of diagnosis to adulthood.Dr Anne-Marie Childs is Consultant Paediatric Neurologist at Leeds Teaching Hospitals NHS Trust and is one of the co-lead of SMA Care UK. She introduces SMA Care UK and then discusses respiratory care. She is joined by Lisa Edel, who is a lead member of the Respiratory Neuromuscular Service at Great Ormond Street Hospital.Professor Baranello is Professor of Paediatric Neuromuscular Disorders at Great Ormond Street Hospital and leads the SMA Care UK Hip Working Group on the revision of hip management. He discusses hip management.Dr. Robert Muni-Lofra is Consultant Neuromuscular Physiotherapist and an Honorary Clinical Senior Lecturer at the John Walton Muscular Dystrophy Research Centre, Newcastle University. He leads the SMA Care UK Spine Working Group. Here, he discusses spine management.