Fenfluramine Provides Clinical Benefit in Adults and Children with Dravet Syndrome: Real-World Experience from the European Early Access Program
作者
Adam Strzelczyk,M. Pringsheim,T. Mayer,Tilman Polster,Arnold R. Gammaitoni,Renzo Guerrini,Nicola Specchio,Ángel Aledo‐Serrano,Francesca Darra,António Gil‐Nagel,Sameer M. Zuberi,Amélie Lothe
出处
期刊:Neuropediatrics [Thieme Medical Publishers (Germany)] 日期:2021-10-28
标识
DOI:10.1055/s-0041-1739607
摘要
Background/Purpose: In a recently completed phase 3 program, patients with Dravet syndrome (DS) treated with fenfluramine demonstrated sustained, profound reductions in seizure frequency, prolonged periods of seizure freedom, and improvement in executive functions. The European Early Access Program (EU-EAP) was initiated to allow pre-approval access to fenfluramine.