NICE Approves Zolgensma Gene Therapy for Pre-Symptomatic Treatment of SMA

The UK SMA Newborn Screening Alliance is delighted that Zolgensma™ (Onasemnogene abeparvovec) is now recommended as an option for treating pre-symptomatic SMA in babies aged 12 months and under, who have up to 3 copies of the SMN2 gene. Previously, babies diagnosed symptomatically could only access Zolgensma through a managed access agreement (MAA).

This recommendation is an important development in relation to newborn screening for SMA.