Novartis: FDA approves AveXis' gene therapy Zolgensma

(CercleFinance.com) - AveXis' Zolgensma is the first and only gene therapy for pediatric patients with spinal muscular atrophy to be approved by the FDA, the Novartis unit said on Monday.


The US Food and Drug Administration has approved Zolgensma for the treatment of pediatric patients of under 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 gene.

Zolgensma has been designed to address the genetic root cause of SMA by providing a functional copy of a human gene to halt disease progression with a single, one-time intravenous infusion.

Copyright (c) 2019 CercleFinance.com. All rights reserved.