An activator of voltage-gated K+ channels Kv1.1 as a therapeutic candidate for episodic ataxia type 1
Mauro Pessia, Fatemah Haidar, Ilenio Servettini, Giuseppe Talani, Alfredo Megaro, Maria Dolores Setzu, Francesca Biggio, Michelle Briffa, Luca Gugliemi, Nicoletta Savalli, Francesca Binda, Francis Delicata, Gilles Bru–Mercier, Neville Vassallo, Vittorio Maglione, Ruben J. Cauchi, Alba Di Pardo, Maria Collu, Paola Imbrici, Luigi Catacuzzeno, Riccardo Olcese, Maria Cristina D’Adamo · FENS Forum 2024
Loss–of–function mutations in the KCNA1(Kv1.1) gene cause episodic ataxia type 1 (EA1), a severe neurological disease characterized by cerebellar dysfunction, ataxic attacks, persistent myokymia with painful cramps in skeletal muscles and epilepsy. Precision medicine for EA1 treatment is currently unfeasible, as no drug that can enhance the activity of Kv1.1–containing channels and offset the functional defects caused by KCNA1 mutations has been clinically approved. Here we uncovered that niflumic acid (NFA), a currently prescribed analgesic and anti–inflammatory drug with an excellent safety
ePoster · Neuroscience