ikona 88

TreatWolfram

Project title: An international, randomized, double-blinded, pivotal clinical trial with the aim to evaluate the efficacy and safety of sodium valproate in children and adults with Wolfram syndrome.

Project description

Presented study is a non-commercial, international, randomised, double-blinded, pivotal study which aim is to show that sodium valproate, currentrly used in the treatment of epilepsy in children and adults, is safe and effective, in slowing the progression of the disease in paediatric and adult patients with Wolfram syndrome.

Coordinating Principal Investigator

prof. Timothy Barrett, MD, PhD

UMED Site Principal Investigator

prof. Wojciech Młynarski, MD, PhD

Categories:

  • Genetic neurodegenerative diseases
  • Rare diseases