SMA Europe is a non-profit umbrella organisation of spinal muscular atrophy (SMA) patient organisations across Europe.

SMA UK is an active member of SMA Europe, with two delegates working closely with colleagues from 24 different European SMA patient groups. We work together to bring effective treatments and optimal care to everyone.

SMA Europe’s priorities:

Research: Work closely with industry to promote patient relevant SMA research.

Therapy and Care:  Accelerate progress in the diagnosis, treatment and care of all those living with SMA.

Healthcare System, Policy and Access: Improve access to diagnosis, optimal treatment and care for all SMA patients in the UK and Europe.

For more information, click on the links below or visit their website.

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