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Journal article : Review

Therapeutic strategies for congenital myasthenic syndromes

Abstract:

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), 3,4-diaminopyridine (3,4-DAP), ephedrine, salbutamol/albuterol, open-channel blockers (fluoxetin...

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Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1111/nyas.13538

Authors


More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Sub department:
Clinical Neurosciences
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Sub department:
Clinical Neurosciences
Oxford college:
St Cross College
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Sub department:
Clinical Neurosciences
Role:
Author
More from this funder
Name:
Medical Research Council
Grant:
MR/M006824/1
Publisher:
Wiley
Journal:
Annals of the New York Academy of Sciences More from this journal
Volume:
1412
Issue:
1
Pages:
129-136
Place of publication:
United States
Publication date:
2018-01-30
Acceptance date:
2017-10-02
DOI:
EISSN:
1749-6632
ISSN:
0077-8923
Pmid:
29381222

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