Description: MLD Gene Therapy - EMA approved, compassionate access, what is gene therapy, clinical trials, and regulatory approval updates
The Premier Family Resource for Metachromatic Leukodystrophy Information & Support since 1999!
Gene therapy is based on the principle that every illness caused by a gene defect can be cured by inserting a corrected (functional) copy of the gene into the sick cells of the patient.
In the case of the MLD, it is difficult for the corrected cells to access the central and peripheral nervous system due to the Blood-Brain-Barrier which works to prevent foreign substances, including vectors carrying corrected genes, from crossing into the central nervous system (brain) and peripheral nerves.