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Novartis shares positive results for spinal muscular atrophy gene therapy Zolgensma

Affecting an estimated one in 10,000 infants globally, SMA is a rare, genetic neuromuscular disease and a leading genetic cause of infant death.

Caused by the lack of a functional SMN1 gene, the most severe forms of SMA result in the rapid and irreversible loss of motor neurons, affecting muscle functions such as breathing, swallowing and basic movement.

Zolgensma is the only gene therapy approved to treat SMA and the only treatment designed to directly address the genetic root cause of the disease.

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