Disease specific therapies in leukodystrophies and leukoencephalopathies

behalf of the GLIA Consortium

Research output: Contribution to journalArticle

21 Citations (Scopus)

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.

Original languageEnglish (US)
Pages (from-to)527-536
Number of pages10
JournalMolecular Genetics and Metabolism
Volume114
Issue number4
DOIs
StatePublished - Apr 1 2015

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Leukoencephalopathies
Patient Care Management
Therapeutics
Phase II Clinical Trials
Clinical Trials, Phase I
Research Personnel
Research

Keywords

  • Care
  • Consensus
  • Leukodystrophy
  • Outcomes
  • Prevention
  • Therapy

ASJC Scopus subject areas

  • Biochemistry
  • Molecular Biology
  • Genetics
  • Endocrinology
  • Endocrinology, Diabetes and Metabolism

Cite this

Disease specific therapies in leukodystrophies and leukoencephalopathies. / behalf of the GLIA Consortium.

In: Molecular Genetics and Metabolism, Vol. 114, No. 4, 01.04.2015, p. 527-536.

Research output: Contribution to journalArticle

behalf of the GLIA Consortium. / Disease specific therapies in leukodystrophies and leukoencephalopathies. In: Molecular Genetics and Metabolism. 2015 ; Vol. 114, No. 4. pp. 527-536.
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