European patient survey reveals ongoing gaps in SMA treatment access
A European survey of people living with spinal muscular atrophy (SMA) has found that while treatment options have expanded since the first medicine was approved in 2017, patient journeys remain far from straightforward.
Data from the 2025 European Patient Experience Survey on SMA Medicines, Access, and Treatment Journeys demonstrated that many patients face inequities at diagnosis; an inability to access, choose, or switch between medicines; and uncertainty with continued treatment.
While existing treatments often manage specific symptoms or slow disease progression, other critical domains may continue to decline, leaving the community hoping for new add-on therapies in the future.
The biannual pan-European survey, the fifth in a series run by the patient organization SMA Europe, gathered evidence from patients, families, and caregivers to better understand their experiences and to guide and inform the organization’s advocacy work.
“People living with SMA and caregivers take an active and fundamental role in the design, piloting, analysis, and interpretation of our surveys,” Alice Larotonda, community research and education manager at SMA Europe, said in an organization press release. “This ensures that the surveys remain focused on patient-relevant questions and that community priorities are genuinely reflected, adding unparalleled value to the evidence generated.”
Survey respondents reflect broad range of disease severity
SMA is a genetic condition in which motor neurons, the nerve cells that control muscle movement, gradually lose function. Left untreated, SMA leads to progressive muscle weakness and loss of physical abilities, and in its most severe forms can affect breathing and swallowing.
In Europe, the first disease-modifying therapy (DMT) for SMA, Spinraza (nusinersen), was approved in 2017. Until early 2025, when survey responses were collected, two more DMTs had become available: Evrysdi (risdiplam), an oral medicine, and Zolgensma (onasemnogene abeparvovec), a one-time gene therapy.
Of the 826 survey respondents from 41 countries, most (58.8%) were people living with SMA, and the remainder were parents or caregivers. Respondents included people with most types of SMA, reflecting a broad range of disease severity.
At diagnosis, 42% said DMTs or clinical trials were available when they or their child was diagnosed. Some families (15%) reported moving to another country to access treatment.
Among those who never started an approved SMA medicine (14.5%), the most common reason was a lack of access, often because of age, genetic, clinical, or functional eligibility criteria.
Nearly 70% reported having no opportunity to choose their medicine
Regarding treatment decisions, most respondents (69.3%) reported having no opportunity to choose their medicine. Still, nearly all felt informed enough to decide whether to start a DMT (85.5%) and which one (87.8%).
Neurologists were the most consulted source of advice (85.5%), followed by other people living with SMA (44.3%) and the internet (38.4%). A majority (58.1%) felt the person with SMA (or their family) and their doctor should make the final decision jointly. Looking back, nearly all (88.5%) said they would make the same treatment decision again.
Participants described starting treatment as both an opportunity (85.3%) and a risk (40.6%). Before starting, about half (55.5%) expected their condition to improve, one-third (34.2%) expected stabilization, and a smaller number (10.3%) expected their condition to worsen despite treatment.
Respondents most often reported improvements in endurance (67.3%), muscle strength (60.9%), and fine motor skills involving the hands, wrists, and fingers to make precise movements (57.3%).
The majority (53% to 71.4%) also reported stabilization of breathing, swallowing, eating, and speaking. Notably, 89.6% of respondents said they consider stabilization, or a lack of disease progression, meaningful progress.
Switching between medicines or adding a second treatment remained relatively uncommon. About one in six respondents (17.5%) had switched between Spinraza and Evrysdi (which have similar mechanisms of action), most often citing administrative or procedural burden (78.2%).
[Treatment] has changed the trajectory of SMA, but not the complexity of living with it.
Among those given Zolgensma, 40% had previously received another DMT, and 17.9% added a DMT after, with most of the latter group hoping for further improvement (62.5%).
Treatment continuity was a concern, with one-fifth (19.5%) reporting that they stopped or interrupted Spinraza or Evrysdi at least once. Nearly half (46.6%) said they did not have enough information about the potential consequences of stopping or interrupting treatment.
Looking forward, half (50.4%) of the respondents feared being forced to stop treatment at some point. Most (78.4%) reported feeling anxious about the possibility that their or their child’s SMA symptoms may get worse in the future.
Despite the benefits of current treatments, nearly all respondents (83.3%) agreed that future SMA medicines are needed. Most (89.2%) wanted therapies that rebuild and strengthen muscle or regenerate motor neurons (86.7%). A majority (78.7%) also felt that SMA patients, with medical advice, should be able to choose which SMA medicine they receive.
The survey shows that treatment “has changed the trajectory of SMA, but not the complexity of living with it.” Calls to action included timely and equitable access to SMA treatments across Europe, informed and personalized treatment decisions, ensuring treatment continuity, and developing add-on therapies targeting multiple needs.
SMA Europe conducted the survey in collaboration with Tilburg University in the Netherlands and with support from the Cure SMA Industry Collaboration.
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