Nusinersen, an exon 7 inclusion drug for spinal muscular atrophy: A minireview

doi:10.13105/wjma.v9.i3.277

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Jun 28, 2021 (publication date) through Feb 22, 2026

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World Journal of Meta-Analysis

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2308-3840

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World J Meta-Anal. Jun 28, 2021; 9(3): 277-285
Published online Jun 28, 2021. doi: 10.13105/wjma.v9.i3.277

Nusinersen, an exon 7 inclusion drug for spinal muscular atrophy: A minireview

Bijaylaxmi Behera

Bijaylaxmi Behera, Department of Neonatology, Chaitanya Hospital, Chandigarh 160044, India

Author contributions: Behera B performed the literature review and wrote the manuscript.

Conflict-of-interest statement: The author has no conflicts of interest and has nothing to disclose.

Corresponding author: Bijaylaxmi Behera, MBBS, MD, Chief Physician, Department of Neonatology, Chaitanya Hospital, Sector 44, Chandigarh 160044, India. jollybubu2008@gmail.com

Received: December 3, 2020
Peer-review started: December 3, 2020
First decision: May 6, 2021
Revised: May 20, 2021
Accepted: June 17, 2021
Article in press: June 17, 2021
Published online: June 28, 2021
Processing time: 221 Days and 2.1 Hours

Abstract

Spinal muscular atrophy is an autosomal recessive neuromuscular disease with incidence of 1 in 5000 to 10000 live births and is produced by homozygous deletion of exons 7 and 8 in the SMN1 gene. The SMN1 and SMN2 genes encode the survival motor neuron protein, a crucial protein for the preservation of motor neurons. Use of the newer drug, Nusinersen, from early infancy has shown improvement in clinical outcomes of spinal muscular atrophy patients.

Keywords: CHERISH; Nusinersen; Spinal muscular atrophy; Survival motor neuron; NURTURE

Core Tip: Spinal muscular atrophy is an autosomal recessive neuromuscular disease, with incidence of 1 in 5000 to 1 in 10000 live births. This review provides an elaborative knowledge regarding the current most effective drug for spinal muscular atrophy, Nusinersen. A brief discussion on other treatment modalities that are under trials is also provided.