New treatments for myasthenia: a focus on antisense oligonucleotides | Zendy

C. Angelini | Zendy; Martignago | Zendy; Bisciglia | Zendy

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New treatments for myasthenia: a focus on antisense oligonucleotides

Author(s) -

C. Angelini,

Martignago,

Bisciglia

Publication year - 2013

Publication title -

drug design development and therapy

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 0.964

H-Index - 64

ISSN - 1177-8881

DOI - 10.2147/dddt.s25716

Subject(s) - acetylcholinesterase , myasthenia gravis , gene isoform , acetylcholine receptor , autoantibody , acetylcholine , aché , pharmacology , medicine , drug , oligonucleotide , chemistry , immunology , endocrinology , receptor , enzyme , antibody , gene , biochemistry

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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