This team of neurologists, located at prestigious hospitals in the United States and Israel, is led by Florian Eichler, Director of the Center for Rare Neurological Disease at Massachusetts General Hospital (MGH). The ML4 Foundation is currently funding a 3-year prospective and retrospective natural history study based at MGH. This study aims to better understand all facets of the disease, better identify the window for intervention, and targets for intervention. The importance of these clinical studies cannot be overstated: only through rigorous clinical studies of patients with MLIV disease can scientists develop the deep knowledge necessary for a treatment that the FDA will approve for use in patients.