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

Summary: Wolfram syndrome is a rare, genetic condition that tragically causes young people to lose their vision and suffer from other neurological issues. Currently, there is no cure to stop this decline. However, researchers are looking into the past to find a future solution. The TREATWOLFRAM trial is investigating whether Sodium Valproate—a medication that has been used for decades to treat epilepsy—can be repurposed to help these patients.

Doctors believe this drug might protect the nerves in the eyes and brain from dying off. In this study, 70 patients are being treated for three years, with some receiving the drug and others a placebo. By measuring changes in their vision, scientists hope to prove that this "old" drug can slow down the disease, offering a new shield against blindness for those living with this difficult syndrome. The trial is currently ongoing, with results expected after late 2024.