August is SMA Awareness Month, and we’re continuing our Every Piece of the Puzzle campaign, celebrating the people, partnerships and milestones that have shaped SMA UK and the wider SMA community. This piece of the puzzle is an overview to securing access to Risdiplam.
The journey to access risdiplam (Evrysdi®) has been shaped by years of research, determination and the voices of people living with spinal muscular atrophy (SMA), their families, clinicians and patient organisations.
In 2026, after 10 years of advocacy and campaigning, the SMA community reached a historic milestone when NICE recommended risdiplam and nusinersen for permanent NHS England funding. The recommendations were finalised on 4 June 2026, recognising the importance of these treatments and the impact they can have on people living with SMA.
But this achievement was built over many years of collaboration and persistence.
Read more detail about access to Risdiplam here
Building the case for access
Risdiplam’s journey began with research and development. In 2017, RG7916 (the original name for risdiplam) was granted orphan drug status in the US, followed by PRIME designation from the European Medicines Agency in 2018.
As the treatment progressed through approval processes, SMA UK, Muscular Dystrophy UK (MDUK) and the wider SMA community worked to ensure that the experiences of people living with SMA were heard.
In 2020, SMA UK and MDUK submitted evidence to NICE, including community surveys highlighting the impact of SMA on daily life and the importance of treatment choices.
While funding decisions were being considered, some people were able to access risdiplam through the Early Access to Medicines Scheme (EAMS). TreatSMA worked successfully with the MHRA to bring this scheme to the UK. They, SMA UK and MDUK then worked to address the many challenges that blocked bringing the EAMS to every regional centre. Each patient organisation levered their different connections to ensure access for every single eligible person. Together with families, clinicians, NHS Trusts and Roche they ensured that 241 people in the UK received treatment before the scheme closed.
The importance of patient voices
The journey through NICE and Scottish Medicines Consortium (SMC) assessments was not always straightforward.
When NICE initially did not recommend risdiplam for NHS funding in 2021, SMA UK and MDUK continued to advocate for the community, ensuring that patient experiences were included in decision-making.
In Scotland, a Patient and Clinician Engagement (PACE) meeting in January 2022 proved an important moment. People living with SMA, clinicians and charities shared the realities of living with SMA and the importance of having access to treatment options.
Following this work, the Scottish Medicines Consortium announced in February 2022 that NHS Scotland would fund risdiplam for eligible people with SMA.
In December 2021, NICE had also issued final guidance recommending risdiplam, marking a major step forward for access in England.
Continuing the campaign for everyone with SMA
Although access improved, the SMA community continued to campaign for permanent and equitable access for everyone living with SMA, regardless of age, SMA type or functional ability.
In 2023, SMA UK , MDUK and Treat SMA responded jointly to NICE’s scoping consultation for the review of risdiplam and nusinersen.
In 2024, SMA UK, working in collaboration with partner organisations, gathered views from the community, receiving responses from 114 people living with SMA and 144 carers. These experiences helped shape a joint patient group submission to NICE, highlighting that treatment benefits are not always fully captured by traditional measures and that real-world experiences must be considered.
When NICE’s review was delayed in 2024, SMA UK continued to work with patient organisations, clinicians and the SMA community to ensure that the full impact of SMA and the benefits of treatment were recognised.
In 2025, further evidence was reviewed, including a new External Assessment Group report and a joint patient organisation response. The focus remained clear: people with SMA should have access to treatments based on individual clinical need, not limited by age, SMA type or other factors.
A landmark achievement
In November 2025, NICE paused its appraisal to review additional evidence around quality of life, healthcare needs and the impact on carers. Patient and clinical voices and the crucial reporting of lived experience, continued to play an important role in ensuring these factors were understood.
Then, in May 2026, the SMA community celebrated a major victory: NICE recommended risdiplam and nusinersen for permanent NHS England funding.
This milestone represents years of dedication from people living with SMA, families, clinicians and advocacy organisations. It shows the power of a community working together to influence change.
The story of risdiplam is not only about access to a medicine, but also about ensuring that the experiences, needs and hopes of people living with SMA are recognised and valued.
