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EAN 2021 | Therapeutic targeting of the safety factor in myasthenic syndromes

Hakan Cetin, MD, PhD, Medical University of Vienna, Vienna, Austria, talks on therapeutic strategies that target the safety factor in myasthenic syndromes. A reduced safety factor, and consequential neuromuscular transmission dysfunction, is a feature of myasthenic syndromes driven by various autoimmune or congenital factors. Determining the nature of the defect is vital to guide the optimal therapeutic approach for an individual. Several agents are available that work to normalize neuromuscular transmission by increasing the safety factor. For example, 3,4-diaminopyridine blocks presynaptic potassium channels to prolong action potentials and increase vesicle release. Pyridostigmine inhibits acetylcholinesterase to increase acetylcholine concentrations in the synaptic cleft. This interview took place during the European Academy of Neurology 2021 congress.