What is GLIA-CTN?

Leukodystrophies are a complex, often progressive group of disorders affecting the white matter of the brain due to the loss or absence of myelin, the lipid membrane that insulates axons in the central nervous system. Despite advances in the diagnosis of these disorders, they remain widely under-recognized, with unmet gaps in clinical care and curative therapeutics.

The Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN) is a consortium of scientists, industry stakeholders, and patient advocacy leaders working together to promote advances in the diagnosis and treatment of leukodystrophy. Specifically, it seeks to create a robust research infrastructure that will allow for the collection and analysis of longitudinal natural history data, development of novel clinical outcome assessments, and identification of surrogate biomarkers – ultimately paving the way for transformative therapeutic trials across the leukodystrophies.

In parallel to these approaches, the GLIA-CTN will work closely with a diverse group of stakeholders to promote disease awareness and education, advocate for the adoption of universal newborn screening and early diagnostic programs, and establish clinical guidelines to support the short- and long-term care of individuals living with leukodystrophies.