Abstract
The effect of 4:1 glyceryl trioleate-glyceryl trierucate (Lorenzo’s oil) on disease progression in 89 asymptomatic boys (mean age 4.7+/-4.1 years; range 0.2-15 years) with X-linked adrenoleukodystrophy (ALD) and normal brain MRI was studied at Johns Hopkins University, Baltimore, MD, and other centers.
Highlights
The effect of 4:1 glyceryl trioleate-glyceryl trierucate (Lorenzo's oil) on disease progression in 89 asymptomatic boys with Xlinked adrenoleukodystrophy (ALD) and normal brain MRI was studied at Johns Hopkins University, Baltimore, MD, and other centers
Thirty-two different ABCD1 mutations were identified by direct sequencing of polymerase chain reaction products in 34 unrelated Chinese X-linked adrenoleukodystrophy (ALD) patients examined at Peking University First Hospital, Beijing, PRC
Mutational analysis of ABCVD1 gene is of value in counseling of ALD families
Summary
The effect of 4:1 glyceryl trioleate-glyceryl trierucate (Lorenzo's oil) on disease progression in 89 asymptomatic boys (mean age 4.7+/-4.1 years; range 0.2-15 years) with Xlinked adrenoleukodystrophy (ALD) and normal brain MRI was studied at Johns Hopkins University, Baltimore, MD, and other centers. Thirty-two different ABCD1 mutations were identified by direct sequencing of polymerase chain reaction products in 34 unrelated Chinese X-linked adrenoleukodystrophy (ALD) patients examined at Peking University First Hospital, Beijing, PRC. Mutational heterogeneity is prevalent in Chinese patients with ALD, a finding consistent with other populations. Mutational analysis of ABCVD1 gene is of value in counseling of ALD families.
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