The UK experience of SMA gene therapy

The UK SMA REACH consortium is reporting its experience in treating infants with SMA with abeparvovec onasemnogene (OA, Zolgensma®):

• 93 babies with type 1 SMA received this treatment between March 2021 and December 2022 at 16 centres across the UK,
• only 75 cases could be accurately documented,
• all had type 1 SMA and were treated at an average age of 11 months, i.e. relatively late, and half of them had already had nusinersen (Spinraza®),
• half of them were able to sit up independently, but almost as many required long-term ventilatory assistance.
• cypho-scoliosis, which was often very asymmetric, was noted in these infants.
• this therapy appears to have been well tolerated overall.

Spinal presentations in children with spinal muscular atrophy type 1 following gene therapy treatment with onasemnogene abeparvovec – The SMA REACH UK network experience. Wolfe A, Sheehan J, Schofield A, et al. Neuromuscul Disord. 2024 Nov.