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Article

Therapeutic strategies for congenital myasthenic syndromes.

Annals of the New York Academy of Sciences - 1 Jan 2018

Lee Manon, Beeson David, Palace Jacqueline

Abstract excerpt

To date, more than 25 genes have been implicated in the etiology of the congenital myasthenic syndromes (CMS), and an ever-growing phenotypic landscape is now encountered in the CMS clinic. Unlike the autoimmune form of myasthenia, there is no role for immunomodulatory agents in the treatment of CMS. The present-day drug repertoire comprises acetylcholinesterase inhibitors (mainly pyridostigmine),...

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Identifiers

PMID: 29381222
DOI: 10.1111/nyas.13538

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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