Sodium valproate, a potential repurposed treatment for the neurodegeneration in Wolfram syndrome (TREATWOLFRAM): trial protocol for a pivotal multicentre, randomised double-blind controlled trial
Renuka P Dias, Kristian Brock, Kun Hu, Rajat Gupta, Una Martin, Andrew Peet, Martin Wilson, Patrick Yu-Wai-Man, Benjamin Wright, Susan Mollan, Archana Kulkarni, Isabelle Meunier, Lucinda Billingham, Zsuzsanna Nagy, Heather Rose, Sulev Koks, Malgorzata Zatyka, Dewi Astuti, Tracy Lynch, Karen E Morrison, Darren Barton, Sabrina Cronier, Rebecca Malpass, Ruth Evans, Amandip Malhi, Pooja Takhar, Amy Lamb, Gema Esteban-Bueno, Wojciech Młynarski, Christophe Orssaud, Agathe Roubertie, Victoria Homer, Timothy Barrett
BMJ Open Feb 2025, 15 (2) e091495; DOI: 10.1136/bmjopen-2024-091495