| Therapy of X-linked adrenoleukodystrophy: prognosis based upon age and MRI abnormality and plans for placebo-controlled trials. | |
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MedLine Citation:
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PMID: 10863943 Owner: NLM Status: MEDLINE |
Abstract/OtherAbstract:
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Evaluation of the therapy of X-linked adrenoleukodystrophy (X-ALD) is hampered by its rarity and by the striking and unpredictable variation in phenotypic expression. We present two approaches that may facilitate therapy evaluation. (1) We have analysed data on 377 X-ALD patients who have been followed at the Kennedy Krieger Institute for a mean period of 38 months and have subdivided them into 18 subgroups on the basis of age and the degree of abnormality in brain magnetic resonance imaging (MRI) as assessed by the Loes score (Am. J. Neuroradrol 1994; 15: 1761). We find that grouping on the basis of age and MRI score provides information that is of significant prognostic value. (2) We present plans for the development of a placebo-controlled multicentre international study that will have sufficient biostatistical power to provide objective evaluation of new therapeutic interventions. |
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Authors:
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H W Moser; L Bezman; S E Lu; G V Raymond |
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Publication Detail:
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Type: Journal Article; Research Support, U.S. Gov't, P.H.S.; Review |
Journal Detail:
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Title: Journal of inherited metabolic disease Volume: 23 ISSN: 0141-8955 ISO Abbreviation: J. Inherit. Metab. Dis. Publication Date: 2000 May |
Date Detail:
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Created Date: 2000-11-09 Completed Date: 2000-11-09 Revised Date: 2007-11-15 |
Medline Journal Info:
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Nlm Unique ID: 7910918 Medline TA: J Inherit Metab Dis Country: NETHERLANDS |
Other Details:
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Languages: eng Pagination: 273-7 Citation Subset: IM |
Affiliation:
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Kennedy Krieger Institute and the Department of Neurology at Johns Hopkins University, Baltimore, MD 21205, USA. moser@kennedykrieger.org |
Export Citation:
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APA/MLA Format Download EndNote Download BibTex |
| MeSH Terms | |
Descriptor/Qualifier:
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Adrenoleukodystrophy
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pathology,
physiopathology* Age Factors Clinical Trials as Topic Humans Magnetic Resonance Imaging Multicenter Studies as Topic Prognosis X Chromosome* |
| Grant Support | |
ID/Acronym/Agency:
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FD-R 000685/FD/FDA HHS; RR00052/RR/NCRR NIH HHS |
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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