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- Guy Helman, Keith Van Haren, Maria L Escolar, and Adeline Vanderver.
- Department of Neurology, Children's National Health System, 111 Michigan Avenue, Northwest, Washington, DC 20010, USA; Center for Genetic Medicine Research, Children's National Health System, 111 Michigan Avenue, Northwest, Washington, DC 20010, USA.
- Pediatr. Clin. North Am. 2015 Jun 1; 62 (3): 649-66.
AbstractThe leukodystrophies are a heterogeneous group of inherited disorders with broad clinical manifestations and variable pathologic mechanisms. Improved diagnostic methods have allowed identification of the underlying cause of these diseases, facilitating identification of their pathologic mechanisms. Clinicians are now able to prioritize treatment strategies and advance research in therapies for specific disorders. Although only a few of these disorders have well-established treatments or therapies, a number are on the verge of clinical trials. As investigators are able to shift care from symptomatic management of disorders to targeted therapeutics, the unmet therapeutic needs could be reduced for these patients.Copyright © 2015 Elsevier Inc. All rights reserved.
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