The 2026 congress represented a milestone for the UK delegation. Representatives from SMA UK, Giles Lomax, Portia Thorman, and Vanessa Christie-Brown,  joined the country’s leading clinicians and researchers to show how clinical studies are improving the daily lives of the SMA community. 

The congress opened with practical sessions designed to solve specific challenges in managing SMA across a person’s whole life. Amongst these workshops were many contributions from the UK.  

1. Interactive Workshops: Focused Care

• Respiratory (Lung) Health: Lisa Edel (UK) co-organised a critical session on proactive lung care. This is essential as new treatments change how respiratory muscles behave over time. 

• Physical Activity (MAPS): José Longatto (Great Ormond Street Hospital) and Marion Main (UK Specialist Physiotherapist) led a workshop on “Mobility, Activity, Play, and Sports,” focusing on how to keep children active at every functional level. 

• Brain and Development: Professor Giovanni Baranello (GOSH) called for international standards to address the cognitive and learning needs that can overlap with SMA. 

• The Research Pipeline: Ewout Groen (University of Edinburgh) led a ‘Bench to Bedside’ session, explaining how a discovery in a laboratory eventually becomes a life-changing treatment for a patient.

2. Main Auditorium: TheWhole BodyApproach 

The main sessions moved beyond the motor neuron, proving that SMA affects the entire body, including blood vessels , metabolism and fatigue.  

• Tracking the Disease: Melissa Bowerman (Keele University) shared how tiny particles released by muscles act as a “Progress Report.” She also presented findings on how Melatonin (a sleep hormone) might help improve symptoms. 

• Nerve-Muscle Connections: Professor Linda Greensmith (UCL) gave a keynote on “optogenetic” therapy, using light to jump-start the connection between nerves and muscles to restore movement. 

• Early Intervention: Charalambos Demetriou (UCL) showed that starting support even before birth (in lab models) could prevent developmental issues before they start. 

• Blood Vessel Health: Alaa Alshwayyat (University of Aberdeen) presented the “EndoSMA” study, proving that SMA affects the tiny blood vessels (microvasculature) that feed our cells, which helps explain why patients feel fatigued. 

• Protein Mistakes: Helena Chaytow (University of Edinburgh) explored how “early mistakes” in how cells build proteins can lead to problems later, helping scientists time treatments more effectively. 

• Rare Types of SMA: Hannah Crick (University of Edinburgh) introduced new ways to study X-linked SMA, a rarer form of the condition. 

• New ways to measure: Researchers introduced new tools, like the SMA EFFORT, to specifically measure perceived physical fatigability (a measure of how quickly someone becomes exhausted from a particular action) in treated patients.  

 3. Setting New Standards of Care

Vanessa Christie-Brown (SMA Care UK) along with leading UK clinicians  presented key findings that will form the rulebook for UK care in the coming years.  

Red more about SMA Care UK’s significant contributions to the congress here. 

4. Advocacy and the Global Stage

Adult Perspectives Workshop (2025): UK’s Luis Castro-E-Canto, who lives with SMA type 2, presented a poster summarising the collaborative work he did with SMA UK. This research gave a global stage to the specific challenges, aspirations, and healthcare gaps faced by adults living with SMA, ensuring their voices are central to future clinical trials and service design.  

Newborn Screening & Treatment Access: SMA UK’s Portia Thorman and Giles Lomax addressed a global audience of advocates, sharing the UK’s strategic journey toward universal screening and sustainable treatment access.  

Our SMArt World: Portia took the opportunity to highlight SMA UK’s children’s book, Our SMArt World. This resource was recognised as a vital tool for representation, helping children and families navigate the complexities of SMA with child friendly explanations, diagrams and illustrations.  

Reflections from Budapest  

“ Returning from the 5th International SMA Scientific Congress in Budapest, I am struck by the immense influence the UK holds on the global stage. It is clear that our delegation is not just participating in these vital conversations, we are leading them.  

What makes me most proud is the deep partnership between SMA UK and our clinical community. Whether it was Vanessa Christie-Brown presenting our findings on hip, spine, and respiratory management , or Luis Castro Canto sharing the essential ‘Adult Perspectives’ from our 2025 workshops, the message was clear: research is only meaningful when it serves the lived experience of people with SMA.  

Our UK clinicians truly understand this. They treat the patient voice as the central guide for their research, ensuring that the new UK Standards of Care are a living commitment to improving daily life. From the main auditorium to the technical workshops, UK experts were at the forefront, proving we are global leaders in this field.  

We leave Budapest with a renewed sense of purpose, knowing that the UK’s clinical engagement is a blueprint for the world. SMA UK will continue to ensure your voice remains at the heart of every future breakthrough.”  

 Portia Thorman Head of Advocacy and Community, SMA UK  

SMA Europe’s 4th International Scientific Congress, Ghent, Belgium

SMA Europe takes great pride in welcoming scientists and young researchers as well as clinicians and other healthcare professionals from all over the world to attend the 4th International Congress on Spinal Muscular Atrophy. The event will bring almost one thousand participants from all over the world.

SMA Europe is the European umbrella for national SMA patient organisations. One of its core activities is to foster patient-relevant research in the field of SMA, to communicate the value generated from research, and consequently to ensure future support for research within our community.

Summary of events

Wednesday 13^th March (Pre-conference events)

• The Global SMAdvocacy Event

This event brings together patient advocates from all over the globe to engage with current issues in SMA patient advocacy. Participants will share advocacy experiences and learn from each other’s strategies.

Discussions will focus on:

• Equity in access to treatments and therapies
• Standards of Care
• Newborn Wcreening
• Influencing policy

Giles Lomax, SMA UK CEO, will be chairing the access to treatments group and Portia Thorman, SMA UK Head of Advocacy and Communtiy, the Newborn Screening group.

• SMA Europe meets early career researchers

SMA Europe is organising an international event to bring together early career researchers and SMA advocates. This will be a unique opportunity for the researchers to engage with the community and to bridge the gap between their laboratory work and the lived experiences of the condition they are working on.

Luis Canto E Castro, SMA Uk’s Community Engagement Lead, will be attending and sharing his personal experiences and those of the UK SMA Community.

Thursday 14^th March

• Is SMA cured?

The future of SMA Research: Panel Discussion.

Session 1 – Rehabilitation in SMA – Motor neuron and spinal cord pathology

It is great to see leading SMA clinicians and academics form the UK presenting in this session: Dr Giovanni Baranello from GOSH, Dr Inga Partlova from Edinburgh University, and Dr Channa Hewamadduma from Sheffield University Hospital.

Session 2 – Should all adults with SMA receive treatment? An experts’ debate

Dr Laurent Servais from The John Radcliffe Hospital, Oxford and Oxford University, will be joined by experts in SMA and ethics from Slovenia and North Macedonia to discuss the ethics of access to treatment for adults.

Flash research presentations:

• Pain in Spinal Muscular Atrophy patients
• Criteria for identification and accurate quantification of spinal motor neurons in healthy and disease mouse models
• Long term changes in compound muscle action potential (CMAP) and their association with motor function in children with infantile-onset SMA
• Monitoring Children with Spinal Muscular Atrophy in Clinical Setting

Session 3 – Optimising gene-directed therapeutics

Chaired by Prof Rafael Yanez-Munoz, Royal Holloway, University of London.

Friday 15^th March

• BIOGEN: Biomarkers of treatment response in SMA.

Session 1 – SMN beyond the motor neuron

Chair: Melissa Bowerman, University of Keele

Session 4 – Developmental aspects of SMA

Chair: Giovanni Baranello, University College London

• NOVARTIS: SMA Journeys: Perspectives from caregivers and clinicians

Flash research presentations:

• Oro facial strength (face and tongue muscles) in symptomatic Type 1 SMA patients treated with nusinersen.
• SMN2 Gene Editing in Spinal Muscular Atrophy
• Disease progression and response to treatment in spinal muscular atrophy
• Electrophysiological assessment of motor unit patterns of the median nerve in adolescents and adults with spinal muscular atrophy
• Biology-based analysis of SMA: Identification of disease relevant protein targets and altered signalling in severe and mild SMA mice.

Session 5- New avenues in SMA research

Chair: Séverine Boilée, Université de la Sorbonne, Paris

Parallel workshops:

Insights from other diseases

Newborn screening

Saturday 16^th March

Session 6 – Muscles in SMA

Chair: Frédéric Relaix, Université de Paris

Presentations will include findings from clinical trials with muscle targeting anti-myostatin drugs, Apitegromab and Taldefgrobep Alfa.

Session 7 – Monitoring disease outcome

Chair: Michelle Farrar, UNSW Sydney, Randwick, Australia