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Spinal muscular atrophy (sma) sma is a rare and devastating genetic disease caused by a lack of a functional survival motor neuron 1 (smn1) gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.

A pair of clinical data sets gives novartis confidence in a new formulation of the company’s gene therapy zolgensma for the treatment of older patients with spinal muscular atrophy (sma) The fda approved itvisma gene therapy for patients aged 2 years and older with spinal muscular atrophy and confirmed smn1 gene mutation. Therapeutic area head of neuroscience at the company. Novartis’ itvisma wins fda approval as the first gene replacement therapy for spinal muscular atrophy (sma) patients age 2 and older. Results from the phase iii steer and phase iiib strength trials show that novartis’ investigational intrathecal gene therapy onasemnogene. Novartis has reported positive topline outcomes from the phase iii steer trial of an investigational gene therapy, intrathecal onasemnogene abeparvovec (oav101 it) in treating individuals with spinal muscular atrophy (sma) type 2