SMA Europe’s 4th Scientific International Congress on Spinal Muscular Atrophy was held between 14 and 16 March, in Ghent, Belgium. It gathered 1,000 participants from 6 continents to further the quest for research in SMA

Written by Emilia Debska, SMA Europe, and Vanessa Christie-Brown, SMA Europe

SMA Europe is an umbrella organisation for spinal muscular atrophy (SMA) patient organisations from across Europe. Currently, it comprises 27 member organisations from 25 countries and keeps growing. The organisation works to bring effective treatments and optimal care to everyone living with SMA.

Research is a fundamental pillar of SMA Europe’s activities and the organisation is committed to promoting patient-relevant SMA research through its projects, funding initiatives and facilitating collaborations. The strategic focus, in 2024, is to prioritise the value generated from research to the SMA community and ensure continued support for future research.

This was the fourth Congress organised by SMA Europe. The previous events took place in Cracow, Poland (2018), Evry, France (2020) and Barcelona (2022). While the number of registrations has grown steadily since the first Congress in Cracow, it is in Ghent where, for the first time, all the seats were sold out and 1,000 participants registered to attend the event.

During the opening speech of the Congress, Nicole Gusset, CEO and president of SMA Europe stated:

“At SMA Europe, one of our goals is to bring stakeholders together and promote dialogue. Originally established to seed-fund fundamental research for rapid therapeutic advances, SMA Europe has evolved to advocate for a partnership role in research.”

“We emphasise the critical importance and benefits of integrating patients into research to understand that research priorities meet patients’ needs.”

– Nicole

Nicole also emphasised the importance of transdisciplinary collaboration to ensure future groundbreaking findings and ensure the quest for progress in the SMA field.

Tom Gillingwater, professor of anatomy at the University of Edinburgh and chair of the scientific advisory board at SMA Europe, acknowledged that the scientific content of the Congress was of the highest standard. He extended a particularly warm welcome to the early career researchers’ community, emphasising that they have the continued support of the rest of the research community. He encouraged the young researchers to be more and more active when pursuing new, groundbreaking ideas in the SMA research field.

The day before the Congress, on 13 March, more than 70 SMA patient advocates, from 50 countries, met during the Global SMAdvocacy event to engage in urgent issues in SMA patient advocacy. This event was held as a kick-off event for the new project led by SMA Europe—SMAcademy, a new educational initiative for SMA patient advocates.

During the meeting, advocates engaged in three workshops to assess the advocacy landscape in SMA, establish end goals and plan strategies to tackle seven challenges in SMA advocacy.

The seven challenges were as follows:

• equity in access to SMA medicines
• reimbursement of SMA medicines
• access and reimbursement of standards of care
• national registries
• access to diagnosis and newborn screening
• supporting SMA-specific research
• influencing policy

The workshops enabled all the patient advocates to contribute their diverse experiences and first-hand expertise to enable all attendees to grow together and move us towards a better future for all people living with SMA.

On the same day, in parallel, the SMA meets Early Career Researchers session was held. This event aimed at fostering the relationship between early career researchers (ECRs) and spinal muscular atrophy (SMA) patient advocates. It was a unique opportunity for ECRs to engage with the broader SMA community and to bridge the gap between their laboratory work and the lived experiences of the rare condition they are working with.

The scientific and clinical programme, designed by the internationally renowned neurologists and scientists who form SMA Europe’s scientific advisory board, brought together the latest advances in SMA academic and healthcare research through a series of talks, workshops and poster presentations.

The programme included 35 oral presentations across 7 topics, chaired by key opinion leaders, 179 posters as well as 4 workshops and sessions with pharmaceutical companies who have clinical research programmes for SMA. There was also a platform for talented early career researchers. Each topic ensured a mix of scientific and clinical research, to ensure a real integration of disciplines.

Topics included:

• Advances in rehabilitation (chaired by Farnaz Nickpour, Liverpool University, UK)
• Optimisation of gene-directed therapeutics (chaired by Rafael Yáñez-Muñoz, Royal Holloway, UK)
• The role of SMN beyond the motor neuron (chaired by Melissa Bowerman, Keele University, UK)
• Developmental aspects of SMA (chaired by Giovanni Baranello, University College London, UK)
• New avenues in SMA research (chaired by Séverine Boilée, Université de la Sorbonne, France)
• Muscles in SMA (chaired by Frédéric Relaix, UPEC, Université de Paris Est Créteil, France)
• Monitoring disease outcome (chaired by Michelle Farrar, University of New South Wales, Australia)

Of particular interest was a workshop held to discuss which high priorities for SMA should lead to a new research agenda.

At the end of the scientific and clinical programme, SMA Europe was delighted to offer a Best Investigator Award, three best poster prizes and two community awards for research that best addresses patient needs.

• The Best Investigator Award: PhD student Sandra Wittig, (Carl Ludwig Institute for Physiology, Germany)
• Best poster prizes: Leandra Ros (UMC Utrecht, Netherlands); Maria Zwartkruis (UMC Utrecht); Maria d’Alaó Gatius Puchercos (IRB Lleida, Universitat de Lleida, Spain)
• Two community awards went to Giorgia Coratti (Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Italy) and Linda-Isabell Schmitt (University Hospital Essen, Germany) for research that best addresses patient needs

The Congress was a huge success that brought the international SMA community closer together and facilitated dialogue among all the stakeholders. Watch out for SMA Europe’s upcoming launch of its 12th Call for Research,which will facilitate and push the most innovative ideas in the field of spinal muscular atrophy. The launch will be announced on  social media and the website.

The 5th Scientific International Congress on Spinal Muscular Atrophy will be held in 2026. The place is still a surprise! 2026 will also be SMA Europe’s 20th Anniversary, so the 5th Congress will acknowledge and celebrate this milestone in its history. A history in which a small group of patient advocates shared their dream of uniting an international community around one goal—to make the world better for all those living with SMA.

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This digital spotlight has been made possible with financial support from F. Hoffmann-La Roche Ltd. The content was developed in collaboration with SMA Europe and F. Hoffmann-La Roche Ltd. All opinions are those of the contributor. RARE Revolution Magazine retains all copyright.

Date of preparation: April 2024 – M-GB-00016467

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