EXPERIENCE ON THERAPY OF ADRENOLEUKODYSTROPHY AND ADRENOMYELONEUROPATHY

被引：43

作者：

UZIEL, G

BERTINI, E

BARDELLI, P

RIMOLDI, M

GAMBETTI, M

机构：

[1] IST NEUROCHIRURG C BESTA,BIOCHIM & GENET SISTEMA NERVOSO LAB,I-20133 MILAN,ITALY

[2] OSPED BAMBINO GESU,REPARTO NEUROPSICHIATRIA INFANTILE,ROME,ITALY

来源：

DEVELOPMENTAL NEUROSCIENCE | 1991年 / 13卷 / 4-5期

关键词：

ADRENOLEUKODYSTROPHY; ADRENOMYELONEUROPATHY; VERY LONG CHAIN FATTY ACIDS; DIETARY TREATMENT; ERUCIC ACID; PEROXISOMAL DISORDER;

D O I：

10.1159/000112173

中图分类号：

Q [生物科学];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

We report the biochemical and clinical results obtained during a dietary erucic acid (C22:1) therapy in 20 patients affected by X-linked adrenoleukodystrophy (ALD). Six patients were very severely affected, 9 had milder neurological symptoms and 5 were presymptomatic. Mean basal levels of plasma C26:0 were 1.41 +/- 0.48-mu-g/ml in ALD patients (control values: 0.33 +/- 0.12). In all patients C26:0 decreased to virtually normal values. In spite of good biochemical response and absence of consistent side effects of therapy, no encouraging data were observed during the clinical follow-up. The presymptomatic subjects were still free of symptoms after more than 1 year of therapy. The symptomatic patients, however worsened or did not show any improvement.

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页码：274 / 279

页数：6

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