Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder caused by mutations in the survival of motor neuron 1 (<i>SMN1</i>) gene, which leads to a reduced level in the SMN protein within cells. Patients with SMA suffer from a loss of alpha motor neurons in the spinal cord l...

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Bibliographic Details

Published in:	Pharmaceutics
Main Authors:	Charlotte A. René, Robin J. Parks
Format:	Article
Language:	English
Published:	MDPI AG 2023-06-01
Subjects:	gene therapy onasemnogene abeparvovec neuromuscular disease motor neuron disease spinal muscular atrophy
Online Access:	https://www.mdpi.com/1999-4923/15/6/1764