VP.54 Amifampridine safety and efficacy in spinal muscular atrophy ambulatory patients: a randomized placebo-controlled, crossover, phase 2 trial

Neuromuscular Disorders - Tập 32 - Trang S92 - 2022
S. Bonanno1, R. Giossi1, R. Zanin1, V. Porcelli1, C. Iannacone2, G. Baranello3, G. Ingenito4, S. Iyadurai4, Z. Stevic5, S. Peric5, L. Maggi1
1Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy
2SPARC Consulting, Milan, Italy
3Dubowitz Neuromuscular Centre, London, UK
4Catalyst Pharmaceuticals, Coral Gables, USA
5University of Belgrade, Belgrade, Serbia