- say stabilisation of disease is a success
Real people, real data
Our European Patient Expectation Survey (EUPESMA) Series are designed to map the experiences and to ascertain the needs and wants of people living with SMA, to help SMA Europe advocate for equal access to optimal treatment and care in Europe.
Key data EUPESMA-2019
- responses in 2019
- no access to medicines
- felt frustration, helplessness or unfairly treated
Demography and access
- 56% had a scoliosis
- contracture were common: 63% in knees, 40% in hips, 32% in elbows, 21% in jaws and almost 20% in wrists and shoulders
- 69% had no breathing assistance / 19% of those who did had a tracheostomy
Snapshot of EUPESMA-2019
Participation by mobility levels and type
1474 people affected by SMA, with both unique and shared experiences, with all SMA types and mobility levels across 26 European countries participated
We have featured a snapshot of the results on this page, please click the EUPESMA-2019 Patient Summary for a full overview of the project.
Why do we need Pan-European patient surveys?
- Access to SMA treatment and care is fragmented across Europe
- Medicines access across different subgroups of people living with SMA is unequal.
- A common Pan European voice is impactful to inform stakeholders about our experiences and also needs, and will eventually help to facilitate change.
- Our previous surveys and especially also EUPESMA-2019 survey showed unequal treatment access across different age groups, SMA types and mobility levels, with the trends between access and either type or mobility severity level lacking in alignment. Based on these outcomes, SMA Europe shaped new projects such as OdySMA and DaLy.
An SMA Europe series
Our SMA patient expectations surveys
Our 1st survey
Our first survey, published in 2017, sought to obtain a view on the clinical state of European people living with Type II and Type III SMA, the impact of this situation on their quality of life and their expectations regarding clinical development.
Our second survey, published in 2021, sought to understand the treatment expectations of people living with SMA after the approval of the first therapy, the realities of daily living and access to clinical trials and therapy, and how this varied according to parameters such as age and disease severity.
- EUPESMA - 2019 Patient Summary
- 2021 - Understanding European patient expectations towards current therapeutic development in spinal muscular atrophy
- 2021 - Impact of Fatigue, Pain, and Psychological Problems on Perceived Quality of Life in Pediatric SMA Patients
- 2017 - Disease impact on general well-being and therapeutic expectations of European Type II and Type III spinal muscular atrophy patients