Hematopoietic stem cell transplantation for the treatment of childhood cerebral X-linked adrenoleukodystrophy: Commentary

Nature Clinical Practice Neurology

Volumn 4, Issue 3, 2008, Pages 130-131

  Maccotta, Luigi ^a   Sherr, Elliott H ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

LORENZO OIL;

ADRENOLEUKODYSTROPHY; CHILDHOOD DISEASE; CLINICAL FEATURE; DISEASE COURSE; DISEASE SEVERITY; FOLLOW UP; GENE MUTATION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; PRIORITY JOURNAL; SHORT SURVEY; SURVIVAL RATE; SURVIVAL TIME; X CHROMOSOME LINKED DISORDER;

ADRENOLEUKODYSTROPHY; COHORT STUDIES; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; MAGNETIC RESONANCE IMAGING; PEDIATRICS; RETROSPECTIVE STUDIES; SEVERITY OF ILLNESS INDEX;

EID: 40549144022     PISSN: 1745834X     EISSN: 17458358     Source Type: Journal    
DOI: 10.1038/ncpneuro0729     Document Type: Short Survey

References (5)

1. Moser H et al. (1995) X-linked adrenoleukodystrophy. In The Metabolic and Molecular bases of Inherited Disease, edn 7 2325-2349 (Eds Scriver C et al.). New York: McGraw-Hill
2. Aubourg P et al. (1990) Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation. New Engl J Med 322: 1860-1886
3. Peters C et al. (2004) Cerebral X-linked adrenoleukodystrophy: The international hematopoietic cell transplantation experience from 1982 to 1999. Blood 104:881-888
4. Moser HW et al. (2005) Follow-up of 89 asymptomatic patients with adrenoleukodystrophy treated with Lorenzo's Oil. Arch Neurol 62 1073-1080
5. Hubbard WC et al. (2006) Combined liquid chromatography-tandem mass spectrometry as an analytical method for high throughput screening for X-linked adranoleukodystrophy and other peroxisomal disorders: preliminary findings. Mol. Genet Metab 89: 185-187.