日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase-Associated Neurodegeneration

泛酸磷酸酯治疗泛酸激酶相关神经退行性疾病的随机对照试验

Klopstock, Thomas; Videnovic, Aleksandar; Bischoff, Almut Turid; Bonnet, Cecilia; Cif, Laura; Comella, Cynthia; Correa-Vela, Marta; Escolar, Maria L; Fraser, Jamie L; Gonzalez, Victoria; Hermanowicz, Neal; Jech, Robert; Jinnah, Hyder A; Kmiec, Tomasz; Lang, Anthony; Martí, Maria J; Mercimek-Andrews, Saadet; Monduy, Migvis; Nimmo, Graeme A M; Perez-Dueñas, Belen; Pfeiffer, Helle Cecilie Viekilde; Planellas, Lluis; Roze, Emmanuel; Thakur, Nivedita; Tochen, Laura; Vanegas-Arroyave, Nora; Zorzi, Giovanna; Burns, Colleen; Greblikas, Feriandas

The FOsmetpantotenate Replacement Therapy (FORT) randomized, double-blind, Placebo-controlled pivotal trial: Study design and development methodology of a novel primary efficacy outcome in patients with pantothenate kinase-associated neurodegeneration

FOsmetpantotenate替代疗法(FORT)随机、双盲、安慰剂对照关键性试验:针对泛酸激酶相关神经退行性疾病患者的新型主要疗效终点的研究设计和开发方法

Klopstock, Thomas; Escolar, Maria L; Marshall, Randall D; Perez-Dueñas, Belen; Tuller, Sarah; Videnovic, Aleksandar; Greblikas, Feriandas