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The 1st International Clinical Care Symposium on SMA brought together nearly 200 participants from around the world. From powerful patient testimonies to compelling science, the message was clear: Focus cannot only be on medicines alone, but also on care. This was a first step. And more will follow. Stay tuned. Thank you all for making history with us.

Medical Emergencies in SMA: pathophysiology, prevention, and response.

SMA experts will be invited to: 1. Present and review the current understanding of the biology of SMA and its impact on the onset of critical episodes; 2. Review state-of-the-art procedures currently in place for SMA in different countries to prevent and manage those emergencies; 3. Align on best practices for managing acute events in SMA.
Main sessions will focus on the biology, clinical presentations and medical management of:&nbsp;
1. Respiratory system in SMA
2. Metabolic system in SMA
3. Bulbar function in SMA (e.g., swallowing pathophysiology, choking, aspiration-related illness)
An additional session will explore experiences from various countries to identify best practices and highlight existing gaps in the prevention and management of critical events.
A broader range of emergency-related areas in SMA will be addressed through a poster session and selected short talks. Abstract submissions are welcome from all in-person attendees.&nbsp;
This will be followed by a practical workshop, showcasing the handling of real-world case examples, and an invitation-only workshop for SMA key opinion leaders to develop conclusions and align on best practices.&nbsp;

Agenda

Symposium25_Programme_June25_final

Friday 29th August 2025
13.15 &ndash; 14.00 Opening (session also streamed live)
&nbsp; &nbsp; &nbsp; &nbsp;Speakers: Dr. Nicole Gusset, SMA Europe CEO; Dr. Yasemin Erbas, SMA Europe President
&ldquo;Patient advocates: Testimony about acute events&rdquo;
&nbsp; &nbsp; &nbsp; &nbsp;Speakers: Stefan Bos, SMA Europe; La&euml;titia Ouillade, AFM T&eacute;l&eacute;thon
Opening Remarks by the Chairs of the Medical Panel:&nbsp; &nbsp; &nbsp; &nbsp; &nbsp;Prof. Stefania Corti, Fondazione IRCCS Ca' Granda Ospedale Maggiore&nbsp; &nbsp; &nbsp; &nbsp; &nbsp;Policlinico, Italy; Prof. Valeria Sansone, Centro Clinico NeMO, Italy
14.00 &ndash; 15.30 Session 1 (session also streamed live)
&nbsp; &nbsp; &nbsp; &ldquo;Respiratory insufficiency in SMA: physiology, prevention and management&rdquo;
&nbsp; &nbsp; &nbsp; &nbsp;Dr. Esther Veldhoen, University Medical Center Utrecht, Netherlands (Keynote speaker)
&nbsp; &nbsp; &nbsp; &nbsp;Prof. Claudio Bruno, Istituto Giannina Gaslini, Italy;
&nbsp; &nbsp; &nbsp; &nbsp;Ms. Lisa Edel, Great Ormond Street Hospital for Children, UK;
&nbsp; &nbsp; &nbsp; &nbsp;Ms. Femke Vercoelen, University Medical Center Utrecht, Netherlands.
15.30-16.00 Coffee Break
16.00 &ndash; 17.30 Session 2 (session also streamed live)
&nbsp; &nbsp; &nbsp; &ldquo;Metabolic disturbances in SMA: physiology, prevention and management&rdquo;
&nbsp; &nbsp; &nbsp; &nbsp;Prof. Stefania Corti, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Italy (Keynote speaker)
&nbsp; &nbsp; &nbsp; &nbsp;Ms. Fay-Lynn Asselman, University Medical Center Utrecht, Netherlands
&nbsp; &nbsp; &nbsp; &nbsp;Prof. Simona Bertoli, DeFENS, University of Milan - IRCCS Istituto Auxologico Italiano, Italy
&nbsp; &nbsp; &nbsp; &nbsp;Dr. Lydia Forestier-Zhang, The University of Sheffield, UK
17.30 &ndash; 18:30 Poster session
18.30 &ndash; 20.00 Networking dinner
Saturday 30th August 2025
08.30 &ndash; 09.00 Session 3 (session also streamed live)
&nbsp; &nbsp; &nbsp;&ldquo;Bulbar system in SMA: physiology, prevention and management&rdquo;
&nbsp; &nbsp; &nbsp; Keynote speaker: Ms. Emily Johnson, Great Ormond Street Hospital for Children, UK
09.00 &ndash; 11.00 Session 4 (session also streamed live)
&nbsp; &nbsp; &nbsp; &ldquo;Country experiences: best practices and existing gaps in managing critical events&rdquo;
&nbsp; &nbsp; &nbsp; &nbsp;Speakers:
&nbsp; &nbsp; &nbsp; &nbsp;Prof. Francesco Muntoni, Dubowitz Neuromuscular Centre (UK)
&nbsp; &nbsp; &nbsp; &nbsp;Dr. Mirella Gaboli, Hospital Universitario Virgen del Roc&iacute;o (Spain)
&nbsp; &nbsp; &nbsp; &nbsp;Prof. Thomas Sejersen, Karolinska Institutet (Sweden)
&nbsp; &nbsp; &nbsp; &nbsp;Prof. Susana Quijano-Roy, H&ocirc;pital Raymond Poincar&eacute; (France)
&nbsp; &nbsp; &nbsp; &nbsp;Prof. Anna Kostera-Pruszczyk, Medical University of Warsaw (Poland)
&nbsp; &nbsp; &nbsp; &nbsp;Prof. Claudio Bruno, Istituto Giannina Gaslini (Italy)
11.00 &ndash; 11.15 Closing Remarks
11.15 - 11.45 Coffee Break
11.45 - 13.15 Practical Workshop (in-person only)
&nbsp; &nbsp; &ldquo;Emergencies encountered in Physiotherapy&rdquo;
&nbsp; &nbsp; &nbsp;Speakers: Dr. Charlotte Lilien, University of Oxford, UK; Ms. Lisa Edel, Great Ormond&nbsp;Street Hospital for Children, UK
&nbsp; &nbsp; &nbsp;&ldquo;Home-Based Emergency Scenarios in Children with SMA Type 1 Before and After Pharmacologic Treatment: The Role of Early Parental Empowerment&rdquo; (for patient advocates)
&nbsp; &nbsp; &nbsp; Speaker: Ms. Chiara Mastella, SAPRE- UONPIA Fondazione IRCCS Ca' Granda&nbsp;Ospedale Maggiore Policlinico Milano, Italy
In parallel:
11.45- 16.00 Final Workshop (by invitation only)
&nbsp; &nbsp; &nbsp;&ldquo;Open discussion to draw conclusions on the addressed topics&rdquo;

Full Programme

MEDICAL PANEL
Stefania Corti (Co-chair)
Neurologist, Full Professor of Neurology and Principal Investigator of Neural Stem Cell Lab at the University of Milan, Italy.
Valeria Sansone (Co-chair)
Clinical and Scientific Director of the NeMO Clinical Center Milan, and Full professor of Neurology at the Department of Biomedical Sciences for Health at the University of Milan.
Melissa Bowerman
Senior Lecturer in Neuromuscular &amp; Skeletal Disorders, School of Medicine, Keele University, UK.
Claudio Bruno
Head, Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genova, and Contract Professor in Paediatrics at the School of Medicine, University of Genova, Italy.
Peter Claus
Group Leader and Professor at the institute of Neuroanatomy and Cell Biology at the Hannover Medical School, Germany.&nbsp;
Ewout Groen
Senior Researcher, SMA Center of Expertise, UMC Utrecht Brain Center, Department of Neurology and Neurosurgery, University Medical Center Utrecht, The Netherlands.&nbsp;
Ulrika Kreicberg
Professor of Palliative Care for Children and Young People at the Great Ormond Street Institute of Child Health, University College London, UK.&nbsp;
C&eacute;cile Martinat
Head of INSERM/UEVE UMR 861 in I-STEM, French institute dedicated to the use of human pluripotent stem cells to study and treat monogenic diseases, Paris, France.&nbsp;
Charlotte Sumner
Professor of neurology at Johns Hopkins School of Medicine, USA.&nbsp;&nbsp;
Ludo van der Pol
Neurologist and Professor of Neurology, head of the Netherlands SMA center at the University Medical Center Utrecht (UMCU) in The Netherlands.&nbsp;
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ORGANIZING COMMITTEE
Yasemin Erbas, PhD, SMA Europe President&nbsp;
Olga Germanenko, SMA Europe Vice-President
Stefan Bos, SMA Europe Board member&nbsp;
Dr. Nicole Gusset, SMA Europe CEO&nbsp;
Federica Fontana, PhD, SMA Europe Research and Medical Manager
Emilia Debska, SMA Europe Communications and Marketing Manager
Theo Soultanos, SMA Europe Operations Manager
Carolina Pachecoy, SMA Europe Operations Support Manager

Faculty

Discounted room rates are available for conference attendees who reserve their room before May 10th.&nbsp;
The venue and the meeting room are fully accessible. The hotel offers eight fully accessible rooms.

Location

1st International Clinical Care Symposium on SMA Supporters

Thanks to our sponsors for their support!
Gold partner - Scholar Rock Silver partners - Novartis - Roche Bronze partner - NMD Pharma Supporting partners - argenx - Biogen

Abstract requirements

Abstract submission

All in-person attendees are invited to submit an abstract.&nbsp;
While the main sessions will focus on metabolic, respiratory, and bulbar systems, as well as experiences from different countries in managing emergencies, abstracts are welcome on any emergency-related aspect of SMA (including fundamental research across different domains, clinical presentations of acute episodes, practical ways of preventing or managing emergencies). Examples of further relevant areas may include cardiovascular, skeletal, gastrointestinal and urological systems, and anaesthetic precautions.
Submitted abstracts are reviewed by the Medical Panel.
Posters will be displayed throughout the Symposium. The presenting author should be present at the poster during the entire duration of the poster session.&nbsp;

1st International Clinical Care Symposium on SMA

Medical Emergencies in SMA: pathophysiology, prevention, and response.

Agenda

Full Programme

Faculty

Location

Sponsors

Abstracts & Posters