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Unlike the autoimmune form of myasthenia, there is no role for immunomodulatory agents in the treatment of CMS. The present\u2010day drug repertoire comprises acetylcholinesterase inhibitors (mainly pyridostigmine), 3,4\u2010diaminopyridine (3,4\u2010DAP), ephedrine, salbutamol\/albuterol, open\u2010channel blockers (fluoxetine, quinidine), or a combination of these. These are prescribed by the specialist in an off\u2010label manner, as there is no drug currently licensed for the treatment of these rare diseases. The effective pharmacological agent varies according to the genetic form of CMS, and it is important to realize that an agent that provides benefit in one CMS subtype can be harmful in another. In addition, the time to treatment response is variable and tends to be commensurate with the drug used. Here, we summarize for the clinician the therapeutic strategies employed in this ever\u2010evolving disease spectrum. We also address the barriers to treatment and discuss the treatment of CMS in pregnancy.<\/jats:p>","DOI":"10.1111\/nyas.13538","type":"journal-article","created":{"date-parts":[[2018,1,30]],"date-time":"2018-01-30T13:14:00Z","timestamp":1517318040000},"page":"129-136","source":"Crossref","is-referenced-by-count":62,"title":["Therapeutic strategies for congenital myasthenic syndromes"],"prefix":"10.1111","volume":"1412","author":[{"given":"Manon","family":"Lee","sequence":"first","affiliation":[{"name":"Nuffield Department of Clinical Neurosciences John Radcliffe Hospital  Oxford United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Beeson","sequence":"additional","affiliation":[{"name":"The Weatherall Institute of Molecular Medicine, John Radcliffe Hospital University of Oxford  Oxford United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jacqueline","family":"Palace","sequence":"additional","affiliation":[{"name":"Nuffield Department of Clinical Neurosciences John Radcliffe Hospital  Oxford United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2018,1,30]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jneuroim.2008.05.030"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.coph.2004.12.007"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1136\/practneurol-2012-000404"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nurt.2008.07.003"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1007\/BF00970931"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp.54.12.1069"},{"key":"e_1_2_9_8_1","first-page":"341","article-title":"Update on treatment options for Lambert\u2013Eaton myasthenic syndrome: focus on use of amifampridine","volume":"7","author":"Lindquist S.","year":"2011","journal-title":"Neuropsychiatr. 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