Disease Specific Therapies in Leukodystrophies and Leukoencephalopathies

Details

• Alternative Title:
  Mol Genet Metab
• Personal Author:
  Helman, Guy ; Van Haren, Keith ; Bonkowsky, Joshua L. ; Bernard, Genevieve ; Pizzino, Amy ; Braverman, Nancy ; Suhr, Dean ; Patterson, Marc C. ; Fatemi, S. Ali ; Leonard, Jeff ; van der Knaap, Marjo S ; Back, Stephen A. ; Damiani, Stephen ; Goldman, Steven A. ; Takanohashi, Asako ; Petryniak, Magdalena ; Rowitch, David ; Messing, Albee ; Wrabetz, Lawrence ; Schiffmann, Raphael ; Eichler, Florian ; Escolar, Maria L. ; Vanderver, Adeline
• Description:
  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.
• Subjects:
  Brain Diseases Demyelinating Diseases Hereditary Central Nervous System Demyelinating Diseases Humans Leukodystrophy, Metachromatic Leukoencephalopathies
• Source:
  Mol Genet Metab. 2015; 114(4):527-536
• Pubmed ID:
  25684057
• Pubmed Central ID:
  PMC4390468
• Document Type:
  Journal Article
• Funding:
  NIH DP2 MH100008/DP/NCCDPHP CDC HHSUnited States/ ; DP2 MH100008/MH/NIMH NIH HHSUnited States/ ; U54NS065768-02/NS/NINDS NIH HHSUnited States/ ; U54 NS065768/NS/NINDS NIH HHSUnited States/ ; R01 MH104701/MH/NIMH NIH HHSUnited States/ ; R01 NS075345/NS/NINDS NIH HHSUnited States/ ; K08 NS060695/NS/NINDS NIH HHSUnited States/ ; R01 MH099578/MH/NIMH NIH HHSUnited States/ ; Z99 HG999999/Intramural NIH HHSUnited States/ ; Canadian Institutes of Health ResearchCanada/ ; 1K08NS060695/NS/NINDS NIH HHSUnited States/
• Volume:
  114
• Issue:
  4
• Collection(s):
  CDC Public Access
• Main Document Checksum:
  urn:sha256:ca0579118fbdf2736fc901d7325abc88f67e7e6a2f59f170b28601b00ca788e2
• Download URL:
  https://stacks.cdc.gov/view/cdc/31309/cdc_31309_DS1.pdf
• File Type:
  [PDF - 258.66 KB ]