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Disease specific therapies in leukodystrophies and leukoencephalopathies.
Helman, Guy; Van Haren, Keith; Bonkowsky, Joshua L; Bernard, Genevieve; Pizzino, Amy; Braverman, Nancy; Suhr, Dean; Patterson, Marc C; Ali Fatemi, S; 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.
Afiliação
  • Helman G; Department of Neurology, Children's National Health System, Washington, DC, USA.
  • Van Haren K; Department of Neurology, Lucile Packard Children's Hospital and Stanford University School of Medicine, Stanford, CA, USA.
  • Bonkowsky JL; Department of Pediatrics and Neurology, University of Utah School of Medicine, Salt Lake City, UT, USA.
  • Bernard G; Department of Pediatrics, Montreal Children's Hospital/McGill University Health Center, Montreal, Canada; Department of Neurology and Neurosurgery, Montreal Children's Hospital/McGill University Health Center, Montreal, Canada.
  • Pizzino A; Department of Neurology, Lucile Packard Children's Hospital and Stanford University School of Medicine, Stanford, CA, USA.
  • Braverman N; Department of Human Genetics and Pediatrics, McGill University and the Montreal Children's Hospital Research Institute, Montreal, Canada.
  • Suhr D; MLD Foundation, USA.
  • Patterson MC; Department of Neurology, Mayo Clinic, Rochester, MN, USA; Department of Pediatrics and Medical Genetics, Mayo Clinic, Rochester, MN, USA.
  • Ali Fatemi S; The Moser Center for Leukodystrophies and Neurogenetics Service, The Kennedy Krieger Institute, Johns Hopkins School of Medicine, Baltimore, MD, USA.
  • Leonard J; The PMD Foundation, USA.
  • van der Knaap MS; Department of Child Neurology, VU University Medical Center, and Neuroscience Campus Amsterdam, Amsterdam, The Netherlands.
  • Back SA; Department of Pediatrics and Neurology, Oregon Health and Science University, Portland, OR, USA.
  • Damiani S; Mission Massimo Foundation Inc., Melbourne, VIC, Australia; Mission Massimo Foundation Inc., Los Angeles, CA, USA.
  • Goldman SA; Center for Translational Neuromedicine and the Department of Neurology of the University of Rochester Medical Center, Rochester, NY, USA.
  • Takanohashi A; Center for Genetic Medicine Research, Children's National Health System, Washington, DC USA.
  • Petryniak M; Department of Pediatrics, Papé Family Pediatric Research Institute, Oregon Health and Science University, Portland, OR, USA.
  • Rowitch D; Department of Pediatrics, University of California, San Francisco, San Francisco, CA, USA; Department of Neurological Surgery, University of California, San Francisco, San Francisco, CA, USA.
  • Messing A; Waisman Center and Department of Comparative Biosciences, University of Wisconsin-Madison, Madison, WI, USA.
  • Wrabetz L; Department of Neurology, Hunter James Kelly Research Institute-HJRKI, University of Buffalo School of Medicine and Biomedical Sciences, Buffalo, NY, USA; Department of Biochemistry, Hunter James Kelly Research Institute-HJRKI, University of Buffalo School of Medicine and Biomedical Sciences, Buffalo
  • Schiffmann R; Institute of Metabolic Disease, Baylor Research Institute, Dallas, TX, USA.
  • Eichler F; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
  • Escolar ML; Department of Pediatrics, University of Pittsburgh, One Children's Hospital Drive, Pittsburgh, PA, USA.
  • Vanderver A; Department of Neurology, Children's National Health System, Washington, DC, USA; Center for Genetic Medicine Research, Children's National Health System, Washington, DC USA; Department of Integrated Systems Biology, George Washington University School of Medicine, Washington, DC, USA. Electronic add
Mol Genet Metab ; 114(4): 527-36, 2015 Apr.
Article em En | MEDLINE | ID: mdl-25684057
ABSTRACT
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.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doenças Desmielinizantes / Doenças Desmielinizantes Hereditárias do Sistema Nervoso Central / Leucoencefalopatias / Leucodistrofia Metacromática Tipo de estudo: Guideline Limite: Humans Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2015 Tipo de documento: Article País de afiliação: Estados Unidos
Texto completo
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Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doenças Desmielinizantes / Doenças Desmielinizantes Hereditárias do Sistema Nervoso Central / Leucoencefalopatias / Leucodistrofia Metacromática Tipo de estudo: Guideline Limite: Humans Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2015 Tipo de documento: Article País de afiliação: Estados Unidos