Technology Prognosis

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Zolgensma (Onasemnogene Abeparvovec) for Spinal Muscular Atrophy

May 29, 2019

Zolgensma (onasemnogene abeparvovec; formerly AVXS-101) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients < 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma is intended to replace the defective SMN1 gene with a functional copy that can create SMN protein, thus preventing the loss of motor neurons and further muscle degeneration in patients with SMA type 1.