The Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN), a research-based consortium for which Children’s Hospital of Philadelphia serves as the lead study site, is working with patient advocates and a network of other institutions to propel research on leukodystrophies toward clinical trial readiness.
This includes a preclinical study demonstrating a novel gene therapy for the treatment of metachromatic leukodystrophy (MLD). Adeline Vanderver a leader of the GLIA-CTN and attending physician at the CHOP Research Institute “We need to continue to work to develop effective and accessible therapies for MLD”.
CHOP news release: Children’s Hospital of Philadelphia Preclinical Study Demonstrates Improved Gene Therapy Approach to Treating Metachromatic Leukodystrophy
Gene therapy research article (March 2025): Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations
