Cargando…
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...
Autores principales: | , , |
---|---|
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 |
_version_ | 1782256105406070784 |
---|---|
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. |
format | Online Article Text |
id | pubmed-3546757 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2013 |
publisher | Dove Medical Press |
record_format | MEDLINE/PubMed |
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 |
work_keys_str_mv | AT angelinicorrado newtreatmentsformyastheniaafocusonantisenseoligonucleotides AT martignagosara newtreatmentsformyastheniaafocusonantisenseoligonucleotides AT biscigliamichela newtreatmentsformyastheniaafocusonantisenseoligonucleotides |