New treatments for myasthenia: a focus on antisense oligonucleotides

reatments for myasthenia: a focus on antisense oligonucleotides Review (1436) Total Article Views Authors: Angelini C, Martignago S, Bisciglia M Published Date January 2013 Volume 2013:7 Pages 13 - 17 DOI: http://dx.doi.org/10.2147/DDDT.S25716 Received: 07 May 2012 Accepted: 31 July 2012 Published: 10 January 2013 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.