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Article

X-linked adrenoleukodystrophy: therapeutic approaches to distinct phenotypes.

Pediatric transplantation - 1 Dec 2005

Mahmood Asif, Dubey Prachi, Moser Hugo W, Moser Ann

Abstract excerpt

X-linked adrenoleukodystrophy (X-ALD) in males can present with eight distinct phenotypes, which vary greatly in respect to phenotypic expression, age of onset and rate of progression and therapy. The plasma very long chain fatty acid assay permits precise diagnosis and is already abnormal at birth. The clinical features, molecular biology, pathogenesis, and therapeutic approaches, including the indications for...

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PMID: 16305618
DOI: 10.1111/j.1399-3046.2005.00447.x

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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