New treatments for myasthenia: A focus on antisense oligonucleotides

IRCCS S Camillo, Via Alberoni, Venice, Italy.
Drug Design, Development and Therapy (Impact Factor: 3.03). 01/2013; 7:13-7. DOI: 10.2147/DDDT.S25716
Source: PubMed


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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