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Current and emerging treatment options for spinal muscular atrophy

Authors Farooq F, MacKenzie A

Received 26 April 2015

Accepted for publication 2 June 2015

Published 17 July 2015 Volume 2015:5 Pages 75—81

DOI https://doi.org/10.2147/DNND.S48420

Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 3

Editor who approved publication: Dr Thomas Müller


Faraz Farooq,1,2 Alex E MacKenzie2,3

1Science Education Division, Emirates College for Advanced Education, Abu Dhabi, United Arab Emirates; 2Children's Hospital of Eastern Ontario (CHEO) Research Institute, Ottawa, ON, Canada; 3University of Ottawa, Ottawa, ON, Canada

Abstract: Spinal muscular atrophy is one of the most common inherited neuromuscular conditions; our understanding of the genetic pathology and translational research coming from this insight has made significant progress over the past decade. This short review provides the background of the disease along with the bench to bedside progress of some promising treatment options to develop better understanding of the present state of the disease.

Keywords: SMN protein, neurodegenerative disease, orphan disease, therapeutics

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