Failure of beta interferon therapy in X-linked adrenoleukodystrophy
β Scribed by G. C. Korenke; H. -J. Christen; D. H. Hunneman; F. Hanefeld
- Publisher
- Springer
- Year
- 1996
- Tongue
- English
- Weight
- 377 KB
- Volume
- 155
- Category
- Article
- ISSN
- 0340-6997
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Five patients with various clinical and genetic phenotypes of adrenoleukodystrophy were treated with a diet enriched with glycerol trioleate and glycerol trierucate (GTE). In all patients platelet counts decreased upon the administration of GTE, but no bleeding symptoms occurred in any of the patien
The clinical course of X-linked adrenoleukodystrophy (X-ALD) is of unexplained heterogeneity. Major X-ALD phenotypes are the progressive childhood cerebral form (CCALD) with early confluent cerebral demyelination and the adult-onset adrenomyeloneuropathy (AMN). Adult AMN may present with demyelinate
In its severe form, X-linked adrenoleukodystrophy (ALD) is a lethal neurologic disease of children, characterized by progressive cerebral demyelination and adrenal insufficiency. Associated with a biochemical defect of peroxisomal β€-oxidation, very long-chain fatty acids (VLCFA) build up in tissues