Clinical trials in myasthenia gravis

Mahmood, A., Hawken, J. and Robertson, N. P. ORCID: https://orcid.org/0000-0002-5409-4909 2023. Clinical trials in myasthenia gravis. Journal of Neurology 270 (9) , pp. 4579-4581. 10.1007/s00415-023-11903-y

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Abstract

Myasthenia Gravis is a chronic autoimmune disease associated with autoantibodies that are directed against post synaptic acetyl choline receptors in 80–88%. Although in many cases initial diagnosis and management is relatively straightforward, myasthenia remains a life-long condition without cure. Current treatment strategies are directed towards symptomatic relief with cholinesterase inhibitors and, in a proportion of patients, suppression of the immune response with the use of steroids and/or longer-term immunosuppressant strategies including oral immunosuppressants, plasma exchange, intravenous immunoglobulin, thymectomy, etc., many of which have significant side effects. Around 50% of patients will not respond to conventional treatment and disease course is often unpredictable with life-threatening myasthenic crises affecting 15–20% of patients at least once during their lives. In addition to the burden of disease on the individual, it has also been estimated that the financial cost from hospital admissions alone as a result of myasthenia was more than €450 million per year in Europe in 2010, and over $500 million in the USA in 2013.

Item Type:	Article
Date Type:	Publication
Status:	Published
Schools:	Medicine
Additional Information:	License information from Publisher: LICENSE 1: URL: http://creativecommons.org/licenses/by/4.0/, Type: open-access
Publisher:	Springer
ISSN:	0340-5354
Date of First Compliant Deposit:	14 August 2023
Date of Acceptance:	26 July 2023
Last Modified:	15 Aug 2023 03:36
URI:	https://orca.cardiff.ac.uk/id/eprint/161715

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