
Current therapies for X-linked adrenoleukodystrophy (X-ALD) include replacement therapy with adrenal steroids, which is mandatory for all patients with impaired adrenal function but does not alter neurological progression significantly; dietary therapy with "Lorenzo's Oil," which appears to have a preventive effect in asymptomatic boys whose brain MRI is normal; and hematopoietic stem cell transplantation in patients in the early stage of the cerebral inflammatory phenotype. Application of these interventions requires careful assessment of the patients' phenotype, which often changes over time. Family screening provides important opportunities for disease prevention.
Drug Combinations, Erucic Acids, Phenotype, Hormone Replacement Therapy, Hematopoietic Stem Cell Transplantation, Humans, Adrenoleukodystrophy, Triolein
Drug Combinations, Erucic Acids, Phenotype, Hormone Replacement Therapy, Hematopoietic Stem Cell Transplantation, Humans, Adrenoleukodystrophy, Triolein
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| influence This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically). | Top 10% | |
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