Novartis's gene therapy Zolgensma (onasemnogene abeparvovec), has been reformulated and shown to benefit to patients aged 2-17 years with spinal muscular atrophy (SMA).
The new formula – called Itvisma – is similar to Zolgensma but is injected directly into the spinal fluid and at a lower viral dose.
SMA is a neurodegenerative condition caused by a lack of survival motor neuron protein encoded by genes including SMN1 and SMN2.
A paper has been published in Nature Medicine detailing the study.
Read more at Inside Precision Medicine.