Disease specific therapies in leukodystrophies and leukoencephalopathies

G. Helman, K. van Haren, J.L. Bonkowsky, G. Bernard, A. Pizzino, N. Braverman, D. Suhr, M.C. Patterson, S.A. Fatemi, J. Leonard, M.S. van der Knaap, S.A. Back, S. Damiani, S.A. Goldman, A. Takanohashi, M. Petryniak, D. Rowitch, A. Messing, L. Wrabetz, R. SchiffmannF. Eichler, M.L. Escolar, A. Vanderver

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39 Citations (Scopus)


Leukodystrophies are a heterogeneous, often progressive group of disorders manifesting a wide range of symptoms and complications. Most of these disorders have historically had no etiologic or disease specific therapeutic approaches. Recently, a greater understanding of the pathologic mechanisms associated with leukodystrophies has allowed clinicians and researchers to prioritize treatment strategies and advance research in therapies for specific disorders, some of which are on the verge of pilot or Phase I/II clinical trials. This shifts the care of leukodystrophy patients from the management of the complex array of symptoms and sequelae alone to targeted therapeutics. The unmet needs of leukodystrophy patients still remain an overwhelming burden. While the overwhelming consensus is that these disorders collectively are symptomatically treatable, leukodystrophy patients are in need of advanced therapies and if possible, a cure.
Original languageEnglish
Pages (from-to)527-536
JournalMolecular Genetics and Metabolism
Issue number4
Publication statusPublished - 2015

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