First baby with SMA in UK receives brand new gene-therapy treatmentKnowledge
A five-month old baby diagnosed with spinal muscular atrophy (SMA) has received life-changing treatment just three weeks after diagnosis.
Arthur Morgan was given a dose of Zolgensma last week, and is thought to be the first infant to receive this potentially curative gene-therapy in the UK. Approved by NICE in March this year, the treatment can enable babies diagnosed with type 1 SMA in the first year of life to reach normal childhood developmental milestones.
Early detection and diagnosis of SMA in babies, followed by rapid access to treatments, can have a huge impact on the quality of life and experiences of both that child and their family. The prescribing of this first dose is a landmark moment in SMA treatment and care.
Access to treatment like Zolgensma, when combined with early detection of the red flags for SMA amongst healthcare professionals like GPs and health visitors, and swift diagnosis by specialists, can transform the lives of the infant with SMA.
The first national paediatric pathway developed for SMA was published last month and provides both a helpful overview, and a detailed roadmap, to optimise detection, diagnosis, treatment and management of SMA - so that more babies like Arthur can receive the life-changing treatment now available.