Behera B. Nusinersen, an exon 7 inclusion drug for spinal muscular atrophy: A minireview. World J Meta-Anal 2021; 9(3): 277-285 [DOI: 10.13105/wjma.v9.i3.277]
Corresponding Author of This Article
Bijaylaxmi Behera, MBBS, MD, Chief Physician, Department of Neonatology, Chaitanya Hospital, Sector 44, Chandigarh 160044, India. jollybubu2008@gmail.com
Research Domain of This Article
Neurosciences
Article-Type of This Article
Minireviews
Open-Access Policy of This Article
This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
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.
Open-Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/Licenses/by-nc/4.0/
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
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.
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.
