Megalencephalic leukoencephalopathy with subcortical cysts (MLC) is type of leukodystrophy (problems in the brain white matter). Most MLC patients carry mutations in the MLC1 gene, encoding a membrane protein of unknown function. We have defined that those mutations affect to the stability of MLC1, leading to its disappearance. In this project our aim is to find compounds that might bind to MLC1 increasing its stability, and therefore increasing its function, which may eventually result in an improvement in the symptoms of MLC patients. These types of compounds have been extremely useful in other conformational diseases such as cystic fibrosis and are being used to treat patients. This project could be the first step to find a pharmacological therapy for MLC patients.