Dr. Ali Fatemi 

Regarding short bio – here excerpt from MLC consortium “”Dr. Ali Fatemi is a pediatric neurologist and the Blum Moser Chair and Chief Medical Officer at Kennedy Krieger Institute and a professor of neurology and pediatrics at Johns Hopkins University. Dr. Fatemi’s career has focused on the study of leukodystrophies and providing care to affected patients” 

Abstract

For decades, leukodystrophies, like many other rare diseases, were neglected in the field of neurology. Most affected individuals lacked an etiologic diagnosis or were diagnosed late in the disease course. However, dramatic progress in neuroimaging and genomics has led to the discovery and early diagnosis of over two hundred genes that affect the central nervous system's white matter. Despite this, the vast majority of these diseases remain untreatable due to their rarity, lack of natural history data, and limited research investments. Over the last decade, several multicenter and multinational consortia have been established to focus on either groups or individual leukodystrophies. These consortia have brought together academic researchers, clinicians, affected patients, patient advocates, and industry partners, accelerating the discovery and development of therapeutics.