New treatments for myasthenia: A focus on antisense oligonucleotides

Corrado Angelini, Sara Martignago, Michela Bisciglia

Research output: Contribution to journalArticle

Abstract

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 signifcant response. The drug is now in a Phase II study. Further investigations are required to confrm its long-term effects. © 2013 Angelini et al, publisher and licensee Dove Medical Press Ltd.

Original languageEnglish
Pages (from-to)13-17
Number of pages5
JournalDrug Design, Development and Therapy
Volume7
DOIs
Publication statusPublished - Jan 9 2013

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Keywords

  • Acetyl cholinesterase
  • Antisense oligonucleotides
  • EN101
  • Myasthenia gravis

ASJC Scopus subject areas

  • Pharmaceutical Science
  • Pharmacology
  • Drug Discovery

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