Sodium valproate, a potential repurposed treatment for the neurodegeneration in Wolfram syndrome (TREATWOLFRAM): trial protocol for a pivotal multicentre, randomised double-blind controlled trial
丙戊酸钠,一种潜在的用于治疗沃尔夫拉姆综合征神经退行性疾病的药物(TREATWOLFRAM):一项关键性多中心、随机、双盲对照试验的试验方案
期刊:BMJ Open
影响因子:2.3
doi:10.1136/bmjopen-2024-091495
Dias, Renuka P; Brock, Kristian; Hu, Kun; Gupta, Rajat; Martin, Una; Peet, Andrew; Wilson, Martin; Yu-Wai-Man, Patrick; Wright, Benjamin; Mollan, Susan; Kulkarni, Archana; Meunier, Isabelle; Billingham, Lucinda; Nagy, Zsuzsanna; Rose, Heather; Koks, Sulev; Zatyka, Malgorzata; Astuti, Dewi; Lynch, Tracy; Morrison, Karen E; Barton, Darren; Cronier, Sabrina; Malpass, Rebecca; Evans, Ruth; Malhi, Amandip; Takhar, Pooja; Lamb, Amy; Esteban-Bueno, Gema; Młynarski, Wojciech; Orssaud, Christophe; Roubertie, Agathe; Homer, Victoria; Barrett, Timothy