Zolgensma (Onasemnogene abeparvovec) is now recommended as an option for treating presymptomatic SMA with up to 3 copies of the SMN2 gene, in babies aged 12 months and under. Previously, babies diagnosed pre-symptomatically could only access Zolgensma through a managed access agreement (MAA).
Many thanks to SMA UK and the clinical and patient experts who, on Thursday 9th February, joined the NICE highly specialised technologies committee meeting to advocate for regular access to Zolgensma for pre-symptomatic babies on the NHS.
This is a crucial decision for the progress of the SMA New Born Screening campaign, -which MDUK are part of through the UK SMA Newborn Screening Alliance. With these important recommendations in place, there will be nothing to delay treatment when the decision to screen for SMA in the UK is finally made.
Read the announcement from NICE in full here.