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