SMA Care UK is a 3-year project launched by clinicians and patient groups to update or develop, promote and implement high-quality care for people of all ages living with spinal muscular atrophy (SMA) across the UK.
As we approach the end of our first year, we’re pleased to share that the project is going very well. All of our first-year goals have been met or are on track.

WHAT IS THE PROJECT ABOUT
We’ve been revising the International Standards of Care (SoC) for SMA (last updated in 2017), with contributions from healthcare professionals (including neurologists, intensivists, respiratory specialists, surgeons, physiotherapists, nurses, care advisors and others) and importantly, people living with SMA.

KEY ACHIEVEMENTS SO FAR

• All Year 1 milestones have been met or exceeded
• A strong leadership team is in place, involving Newcastle University, Leeds Hospitals NHS Trust and SMA UK
• Wide representation across our Steering Committee has been ensured, bringing together professionals and patient voices from across the UK

WHO IS LEADING THE PROJECT?

• Professor Chiara Marini-Bettolo, Consultant Paediatric & Adult Neurologist, Newcastle
• Dr. Anne-Marie Childs, Consultant Paediatric Neurologist, Leeds
• Vanessa Christie-Brown, Project Manager, SMA UK & Newcastle University
• Grecia Benesperi, Project Assistant, Newcastle University

PROGRESS ON NEW CARE GUIDELINES
We began by focusing on four priority areas. Each has a clinical lead working with a working group (WG) that includes expert health professionals and people living with SMA. We’ve also gathered wider input via Facebook discussions, online meetings and workshops.
The four focus areas:

• Respiratory (Breathing) Care
  The WG has reviewed the current SoC. They have discussed and agreed new ideas and practice. They are now writing a new guide. Endorsement is being sought through NICE, The SMA REACH Networks and The British Thoracic Society has approved it in principle.
• Spine Management
  The WG has reviewed the previous SoC. They have discussed and agreed new ideas and practice. These will go to the SMA REACH networks for consensus. Once consensus obtained, the new guidance will be written up and endorsement by the NICE, The British Scoliosis Society and the SMA REACH Networks sought.
• Transition to Adulthood
  A WG has just started meeting and is looking into how best to support young people with SMA move from paediatric to adult services, as they become adults. The group is currently reviewing the research available on this topic.
• Hip Management
  There is no guidance for hip care, so we are writing them from scratch. This WG is using information from studies and advice from experts, such as physiotherapists and surgeons.

SEEKING ENDORSEMENT OF THE GUIDELINES BY PROFESSIONAL BODIES
We are asking NICE and relevant medical societies to endorse the new guidelines so they can:

1. Be credible and have clinical authority: endorsement shows that the guideline is based on robust evidence and expert consensus. It gives clinicians greater confidence to follow the recommendations in routine care.
2. Be integrated into national policy and practice: if NICE curates or endorses the guidance, it becomes more likely to be used as a standard of care across the NHS. NICE-endorsed guidance may be integrated into commissioning frameworks, service specifications, or audit standards.
3. Support funding and resource allocation: commissioners and service planners often require NICE-recognised standards when deciding how to allocate resources. Endorsed guidance can help make the case for funding specialist services, staffing, or equipment.
4. Be adopted by multidisciplinary teams: guidelines endorsed by relevant societies (e.g. BTS for respiratory, BSS for spine) are more likely to be taken up by specialists within those disciplines. It helps drive cross-specialty alignment, especially in complex conditions like SMA.
5. Ensure patient benefit and equity of access: endorsed guidelines help ensure consistent care, regardless of where a patient lives. They can reduce regional differences and promote equitable access to high-quality treatment.
6. Enhance visibility and international collaboration: endorsement by recognised UK bodies enhances the guideline’s visibility and credibility globally, supporting alignment with international standards.

WHAT IS NEXT?
We’ve also started work on new topics based on community and professional feedback:

• Bone health
• Nutrition & swallowing (starting soon)
• A third group, likely Physiotherapy & Exercise, will begin in September

HOW PEOPLE WITH SMA ARE INVOLVED

• Individuals with SMA sit on every working group
• We have Facebook forum where people can share their views and experiences. They are easy to join, follow this link!
• Dedicated online meetings
• We held two dedicated workshops for adults with SMA
• A community session will be held at The Big Weekend
• We are writing patient-friendly versions of each guideline

MEASURING OUR IMPACT
To see what is working in real life, we are creating two national surveys:

• One for health professionals
• One for people living with SMA and their families

These will help us understand:

• Which parts of care are working well
• Where there are gaps or variations across the UK
• How care can be improved#

We will repeat the surveys later to see what’s changed as a result of this project.

WORKING INTERNATIONALLY
We’re working with partners around the world to stay aligned with the best available research and care practices. These include:

• Australia’s SMA clinical network
• Cure SMA (USA)
• European centres

RAISING AWARENESS
We’re sharing our progress at national and international conferences. So far, we’ve been accepted at:

• Association of British Neurologists (Liverpool)
• Cure SMA Conference (USA)
• European Paediatric Neurology Society (Munich)
• World Muscle Society Congress: (Vienna) with 3 presentations

We’ve also submitted abstracts to events in London, Glasgow and Milan.

WHAT’S NEXT (SUMMER–AUTUMN 2025)

• Finalise the first guidelines
• Write easy-to-understand versions for families
• Start the next set of guidelines
• Hold a stakeholder meeting to share updates, adjust plans if needed and address research needs
• Continue working with global and national partners to ensure the guidelines have the widest reach and impact

STAY INVOLVED
Visit the SMA Care UK website for updates, news and information.
If you would like to get involved, contact our Project Manager, Vanessa Christie-Brown at:
• vanessa.christie-brown@smauk.org.uk
• Vanessa.Christie-Brown@newcastle.ac.uk

THANK YOU!
Thank you to everyone in the SMA community who has shared their experiences and insights. Your voices are driving real change in care for everyone with SMA across the UK.