A different formulation of Novartis’s gene therapy Zolgensma (onasemnogene abeparvovec), known as Itvisma, achieved good results in a Phase III trial in spinal muscular atrophy (SMA) patients aged 2-17 years.

While the improvements in mobility are smaller than those seen in younger children treated with Zolgensma, they are still enough to give these children significant improvements in their daily life.

SMA is a rare inherited disease in which a fault in both copies of the SMN1 gene means the body cannot make enough of the protein encoded by this gene, which is needed for healthy motor neuron cells. Over time, these nerve cells die and cannot grow back, causing gradually worsening muscle weakness that impacts breathing, swallowing, and normal movement.

Zolgensma, originally developed

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