Current and emerging treatment options for spinal muscular atrophy

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...

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Detalles Bibliográficos
Autores principales: Farooq, Faraz, MacKenzie, Alex E
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove 2015
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7337203/
https://www.ncbi.nlm.nih.gov/pubmed/32669914
http://dx.doi.org/10.2147/DNND.S48420
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author Farooq, Faraz
MacKenzie, Alex E
author_facet Farooq, Faraz
MacKenzie, Alex E
author_sort Farooq, Faraz
collection PubMed
description 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.
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spelling pubmed-73372032020-07-14 Current and emerging treatment options for spinal muscular atrophy Farooq, Faraz MacKenzie, Alex E Degener Neurol Neuromuscul Dis Review 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. Dove 2015-07-17 /pmc/articles/PMC7337203/ /pubmed/32669914 http://dx.doi.org/10.2147/DNND.S48420 Text en © 2015 Farooq and MacKenzie http://creativecommons.org/licenses/by-nc/3.0/ This work is published by Dove Medical Press Limited, and licensed under Creative Commons Attribution – Non Commercial (unported, v3.0) License. The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. Permissions beyond the scope of the License are administered by Dove Medical Press Limited. Information on how to request permission may be found at: http://www.dovepress.com/permissions.php
spellingShingle Review
Farooq, Faraz
MacKenzie, Alex E
Current and emerging treatment options for spinal muscular atrophy
title Current and emerging treatment options for spinal muscular atrophy
title_full Current and emerging treatment options for spinal muscular atrophy
title_fullStr Current and emerging treatment options for spinal muscular atrophy
title_full_unstemmed Current and emerging treatment options for spinal muscular atrophy
title_short Current and emerging treatment options for spinal muscular atrophy
title_sort current and emerging treatment options for spinal muscular atrophy
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7337203/
https://www.ncbi.nlm.nih.gov/pubmed/32669914
http://dx.doi.org/10.2147/DNND.S48420
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