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New treatments for myasthenia: a focus on antisense oligonucleotides

Autoimmune myasthenia gravis (MG) is a neuromuscular disorder caused by autoantibodies directed against the acetylcholine receptor (AChR). Current symptomatic therapy is based on acetylcholinesterase (AChE) drugs. The available long-term current therapy includes steroids and other immunomodulatory a...

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Detalles Bibliográficos
Autores principales: Angelini, Corrado, Martignago, Sara, Bisciglia, Michela
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove Medical Press 2013
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3546757/
https://www.ncbi.nlm.nih.gov/pubmed/23341732
http://dx.doi.org/10.2147/DDDT.S25716
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author Angelini, Corrado
Martignago, Sara
Bisciglia, Michela
author_facet Angelini, Corrado
Martignago, Sara
Bisciglia, Michela
author_sort Angelini, Corrado
collection PubMed
description Autoimmune myasthenia gravis (MG) is a neuromuscular disorder caused by autoantibodies directed against the acetylcholine receptor (AChR). Current symptomatic therapy is based on acetylcholinesterase (AChE) drugs. The available long-term current therapy includes steroids and other immunomodulatory agents. MG is associated with the production of a soluble, rare isoform of AChE, also referred as the “read-through” transcript (AChE-R). Monarsen (EN101) is a synthetic antisense compound directed against the AChE gene. Monarsen was administered in 16 patients with MG and 14 patients achieved a clinically significant response. The drug is now in a Phase II study. Further investigations are required to confirm its long-term effects.
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spelling pubmed-35467572013-01-22 New treatments for myasthenia: a focus on antisense oligonucleotides Angelini, Corrado Martignago, Sara Bisciglia, Michela Drug Des Devel Ther Review Autoimmune myasthenia gravis (MG) is a neuromuscular disorder caused by autoantibodies directed against the acetylcholine receptor (AChR). Current symptomatic therapy is based on acetylcholinesterase (AChE) drugs. The available long-term current therapy includes steroids and other immunomodulatory agents. MG is associated with the production of a soluble, rare isoform of AChE, also referred as the “read-through” transcript (AChE-R). Monarsen (EN101) is a synthetic antisense compound directed against the AChE gene. Monarsen was administered in 16 patients with MG and 14 patients achieved a clinically significant response. The drug is now in a Phase II study. Further investigations are required to confirm its long-term effects. Dove Medical Press 2013-01-10 /pmc/articles/PMC3546757/ /pubmed/23341732 http://dx.doi.org/10.2147/DDDT.S25716 Text en © 2013 Angelini et al, publisher and licensee Dove Medical Press Ltd. This is an Open Access article which permits unrestricted noncommercial use, provided the original work is properly cited.
spellingShingle Review
Angelini, Corrado
Martignago, Sara
Bisciglia, Michela
New treatments for myasthenia: a focus on antisense oligonucleotides
title New treatments for myasthenia: a focus on antisense oligonucleotides
title_full New treatments for myasthenia: a focus on antisense oligonucleotides
title_fullStr New treatments for myasthenia: a focus on antisense oligonucleotides
title_full_unstemmed New treatments for myasthenia: a focus on antisense oligonucleotides
title_short New treatments for myasthenia: a focus on antisense oligonucleotides
title_sort new treatments for myasthenia: a focus on antisense oligonucleotides
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3546757/
https://www.ncbi.nlm.nih.gov/pubmed/23341732
http://dx.doi.org/10.2147/DDDT.S25716
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