4-Aminopyridine is a promising treatment option for patients with gain-of-function -encephalopathy.
Sanjay M Sisodiya, Holger Lerche, Monika Fudali, Niklas Schwarz, Christian Bosselmann, Pu Yan, Kirsten Torge, Harshad Pannikkaveettil Ashraf, Florian Lutz, Guido Rubboli, Dragan Marjanovic, Lily Wong-Kisiel, Michal Tzadok, Bruria Ben Zeev, Kathleen M Gorman, Mary D King, Nicholas M Allen, Pedro Martínez-Ulloa, José M Serratosa, Ulrike B S Hedrich, Stephan Lauxmann, Markus Wolff, Matthis Synofzik, Thomas Bast, Adrian Binelli
Science translational medicine 2021;13(609):eaaz4957