Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy
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DOI: 10.1080/14712598.2022.2124857
Abstract: ABSTRACT Introduction Adrenoleukodystrophy (ALD) is an X-linked peroxisomal disorder with an incidence of 1 in 14–17,000 male births, caused by pathogenic variants within the ABCD1 gene. By adulthood, approximately 40% of the patients develop cerebral… read more here.
Keywords: therapy; cerebral ald; gene therapy; transplantation ... See more keywords
International Recommendations for the Diagnosis and Management of Patients With Adrenoleukodystrophy
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DOI: 10.1212/wnl.0000000000201374
Abstract: Pathogenic variants in the ABCD1 gene cause adrenoleukodystrophy (ALD), a progressive metabolic disorder characterized by 3 core clinical syndromes: a slowly progressive myeloneuropathy, a rapidly progressive inflammatory leukodystrophy (cerebral ALD), and primary adrenal insufficiency. These… read more here.
Keywords: international recommendations; clinical surveillance; diagnosis management; cerebral ald ... See more keywords