SMA Ireland is pleased to share an important and very welcome development in our ongoing campaign to secure access to treatment for adults living with Spinal Muscular Atrophy in Ireland.
The HSE Drugs Group has recommended in favour of reimbursing risdiplam, also known as Evrysdi, for a defined group of adults with SMA, subject to the establishment of a Managed Access Protocol.
This recommendation relates to adults aged 18 years and older with a clinical diagnosis of SMA Type 2 or Type 3, or with one to four SMN2 copies, who have not previously received a disease-modifying therapy.
This is not yet final approval, and we must be careful not to get ahead of the process. However, it is a very significant milestone.
For many years, adults with SMA in Ireland have been left behind. Since the first SMA medicines became available here, access has been restricted largely on the basis of age. This has created an unfair and arbitrary situation where some adults receive treatment because they were under 18 when access opened, while others with the same condition and similar clinical needs have remained excluded simply because they were already adults at the time.
SMA Ireland has long argued that this is unjust, inequitable and clinically difficult to defend.
SMA is a progressive condition. It does not stop at 18. Adults who are untreated continue to lose strength, independence and function over time. For adults living with SMA, stabilisation can be a deeply meaningful outcome. Preserving the ability to work, study, communicate, use technology, or maintain independence in daily life can make an enormous difference.
This latest recommendation recognises that reality.
The campaign for adult access has involved many people over many years. SMA Ireland would especially like to thank Muscular Dystrophy Ireland, who have been a tremendous partner and ally in the latter stages of this campaign. Their support, advocacy, organisation and commitment helped bring renewed focus and energy to this issue.
We also thank the adults with SMA who shared their stories, spoke publicly, contacted representatives and helped explain the real-life impact of being denied treatment. Their courage and persistence have been central to this progress.
We are also grateful to the clinicians, public representatives and supporters who listened, engaged and helped keep this issue on the agenda.
There are still important steps ahead. A Managed Access Protocol must now be developed, and the final reimbursement process must be completed. SMA Ireland will continue to monitor this closely and will keep the community updated as more information becomes available.
For now, this is a moment of cautious optimism.
It is not the end of the road, but it is the clearest sign yet that the voices of adults with SMA are finally being heard.
Where there’s breath, there’s hope.
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