Zolgensma - One-Time Gene Therapy for Spinal Muscular Atrophy

Date: July 29, 2019
Issue #: 
1577Summary: 
The FDA has approved onasemnogene abeparvovec-xioi (Zolgensma– Avexis), an adeno-associated virus
vector-based gene therapy, for one-time IV treatment
of children<2 years old who have spinal muscular
atrophy (SMA) and bi-allelic mutations in the survival
motor neuron 1 (SMN1) gene. It is the first gene therapy
and the second drug to be approved in the US for SMA;
nusinersen(Spinraza) was approved in 2016.

Source: The Medical Letter - June 27, 2019 Category: Drugs & Pharmacology Authors: admin Tags: nusinersen onasemnogene abeparvovec spinal muscular atrophy Spinraza zolgensma Source Type: research