Presentation description

Vanishing White Matter (VWM) Disease, a rare genetic disorder caused by mutations in the eIF2B complex, critical for protein synthesis regulation. Our research employs a mouse model mimicking human mutations to understand and treat VWM. By uncovering the disease's molecular pathways, we aim to develop safe and effective gene therapy treatments, offering solutions for those affected by VWM worldwide. This endeavor marks a significant step forward in addressing the challenges posed by VWM, bringing hope to patients and their families.