Current and emerging treatment options for spinal muscular atrophy

Faraz Farooq; Alex E MacKenzie

doi:10.2147/DNND.S48420

Current and emerging treatment options for spinal muscular atrophy

Degener Neurol Neuromuscul Dis. 2015 Jul 17:5:75-81. doi: 10.2147/DNND.S48420. eCollection 2015.

Authors

Faraz Farooq^{1

2}, Alex E MacKenzie^{2

3}

Affiliations

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

Publication types

Review