Sodium valproate, a potential repurposed treatment for the neurodegeneration in Wolfram syndrome (TREATWOLFRAM): trial protocol for a pivotal multicentre, randomised double-blind controlled trial

Sodium valproate, a potential repurposed treatment for the neurodegeneration in Wolfram syndrome (TREATWOLFRAM): trial protocol for a pivotal multicentre, randomised double-blind controlled trial

Introduction Wolfram syndrome (WFS1-Spectrum Disorder) is an ultra-rare monogenic form of progressive neurodegeneration and diabetes mellitus. In common with most rare diseases, there are no therapies to slow or stop disease progression. Sodium valproate, an anticonvulsant with neuroprotective prope...

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Main Authors: Una Martin, Timothy Barrett, Susan Mollan, Karen E Morrison, Ruth Evans, Isabelle Meunier, Rajat Gupta, Andrew Peet, Martin Wilson, Sulev Koks, Wojciech Młynarski, Patrick Yu-Wai-Man, Agathe Roubertie, Darren Barton, Dewi Astuti, Renuka P Dias, Lucinda Billingham, Kun Hu, Malgorzata Zatyka, Archana Kulkarni, Benjamin Wright, Kristian Brock, Victoria Homer, Rebecca Malpass, Heather Rose, Christophe Orssaud, Zsuzsanna Nagy, Tracy Lynch, Sabrina Cronier, Amandip Malhi, Pooja Takhar, Amy Lamb, Gema Esteban-Bueno
Format: Article
Language:English
Published: BMJ Publishing Group 2025-02-01
Series:BMJ Open
Online Access:https://bmjopen.bmj.com/content/15/2/e091495.full
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https://bmjopen.bmj.com/content/15/2/e091495.full

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