Advocacy for Access to Treatments
Advocacy for Access to Treatments
We have been advocating for access to treatments in the UK since autumn 2016. This continues to be a high priority. It includes advocacy for access to newborn screening for SMA to enable the earliest possible access to disease-modifying treatments.
Advocacy for Access to
Intrathecal Onasemnogene Abeparvovec (OAV101 IT)
The National Institute for Health and Care Excellence (NICE) is looking at the clinical and cost effectiveness of this treatment. SMA UK and partners are making sure the patient and carer perspective is clearly understood.
NICE recommends nusinersen and risdiplam for permanent NHS England funding
14th May 2026
After 10 years of advocacy and campaigning, SMA UK are delighted to celebrate this hard-won milestones with the SMA Community. NICE’s recommendation ends the dark cloud of anxiety for the future that has been there for so many.
