SMA Europe Report from Warsaw, Poland
24 October 2019
Polish member organisation, Fundacja SMA, were excellent hosts for SMA Europe’s biannual meeting between 10th & 12th October.
SMA Europe’s General Assembly is made-up of Patient Organisations from 19 member countries with up to two delegates from each (for SMA UK - Liz Ryburn, Support Services Manager, and Hassan Sobati, Trustee). It is continuing to expand and grow rapidly across Europe, widening its scope to include more active advocacy for the patient voice across all aspects of SMA.
The General Assembly heard updates on the progress made by its working groups and Patient Advisory Groups (PAGs), who give the patient perspective to industries developing SMA programmes. Two of these companies, Roche and AveXis, gave updates us on their work.
The topic work updates included SMA Europe’s:
1. Latest pan-European patient expectation survey: This is a follow-up from a survey carried out in 2015 (read more here). There has been a big response and now the group is working on analysing the results ready to present them as powerful evidence of what people with SMA have to say about:
- The on-going high need for the development of additional trials with current and new therapeutic approaches for all patients
- The approval of effective and safe therapeutics
- The access to approved therapies
2. Clinical trial readiness project: This is being run in collaboration with Cure SMA and the Industry Consortium in the US. The aim is to understand and expand Europe’s current capacity to run clinical trials for potential SMA therapies. SMA Europe’s 2019 work has involved giving training opportunities to clinical trial centres across Europe, culminating in a best practice workshop led by Dr. Mariacristina Scoto and fellow trainers, Professor Laurent Servais and Dr. Giovanni Baranello, which will take place on 4th February 2020.
3. Patient Reported Outcomes (PROMs): SMA Europe is providing patient input into existing efforts to develop databases and registries that are and will be collecting data about the outcome of treatments. Through this project, SMA Europe intends to ensure the patient's voice and what outcomes matter to people with SMA in their everyday lives is taken into account in the study of disease progression.
4. International scientific & clinical congress on SMA: This takes place between 5th & 7th February 2020 at Généthon, Evry, France. It will welcome scientists, clinicians, industries running SMA clinical trials and patient advocates. SMA Europe is very proud of their exceptional speaker line-up, which includes a talk from Professor Judith Melki who led her team to the discovery of the gene in 1995. To find out more, please visit the congress website.
5. Newborn Screening: SMA Europe is working on a position statement, which will be made available following the publication of the recently held European Neuro Muscular Conference (ENMC) workshop.
In parallel, SMA Europe’s Scientific Advisory Board met to review 19 SMA research grant applications received through their 10th Call for research proposals. More news will follow.
And finally, the Polish Minister of Health, Łukasz Szumowski, came to meet all the delegates and to learn more about the work of SMA Europe, and emphasised his view of the the importance of introducing newborn screening for SMA.