4-Aminopyridine is a promising treatment option for patients with gain-of-function KCNA2-encephalopathy.

Ulrike B S Hedrich Stephan Lauxmann Markus Wolff Matthis Synofzik Thomas Bast Adrian Binelli José M SerratosaPedro Martínez-UlloaNicholas M AllenMary D KingKathleen M Gorman Bruria Ben ZeevMichal TzadokLily Wong-Kisiel Dragan Marjanovic Guido RubboliSanjay M SisodiyaFlorian Lutz Harshad Pannikkaveettil AshrafKirsten TorgePu YanChristian Malte Boßelmann Niklas Schwarz Monika Fudali Holger Lerche

Published in: Science translational medicine (2021)

[Figure: see text].

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