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New treatments for myasthenia: a focus on antisense oligonucleotides

Authors Angelini C, Martignago S, Bisciglia M

Received 7 May 2012

Accepted for publication 31 July 2012

Published 10 January 2013 Volume 2013:7 Pages 13—17


Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 3

Corrado Angelini,1 Sara Martignago,2 Michela Bisciglia2

1IRCCS S Camillo, Via Alberoni, Venice, Italy; 2Department of Neurosciences, University of Padova, Via Giustiniani 5, 35128, Padova, Italy

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 significant response. The drug is now in a Phase II study. Further investigations are required to confirm its long-term effects.

Keywords: myasthenia gravis, antisense oligonucleotides, acetyl cholinesterase, EN101

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