We’re delighted to hear the news today that the first baby in England has been treated with Zolgensma, a potentially life-changing gene therapy for spinal muscular atrophy (SMA) Type 1.
This treatment was delivered at Evelina Children’s Hospital in London, one of four centres in England that will offer the single-dose infusion. This treatment offers significant positive impacts on motor milestones and has the potential to improve the lives of children affected by this rare disease.
Zolgensma is a ground-breaking treatment for children with SMA Type 1. This is the most severe form of SMA, a condition that affects nerve cells in the spinal cord, affecting movement and, sometimes, breathing. Without treatment, babies with SMA Type 1 rarely live past their second birthday.
MDUK has been working with SMA UK, TreatSMA patient groups and clinicians to advocate for access to this innovative treatment. We await further announcements from NICE and will update you about access to this treatment.