[HTML][HTML] X-linked adrenoleukodystrophy (X-ALD): clinical presentation and guidelines for diagnosis, follow-up and management

M Engelen, S Kemp, M De Visser, BM van Geel… - Orphanet journal of rare …, 2012 - Springer
Abstract X-linked adrenoleukodystrophy (X-ALD) is the most common peroxisomal disorder.
The disease is caused by mutations in the ABCD1 gene that encodes the peroxisomal …

[HTML][HTML] X-linked adrenoleukodystrophy: clinical, metabolic, genetic and pathophysiological aspects

S Kemp, J Berger, P Aubourg - … Et Biophysica Acta (BBA)-Molecular Basis …, 2012 - Elsevier
X-linked adrenoleukodystrophy (X-ALD) is the most frequent peroxisomal disease. The two
main clinical phenotypes of X-ALD are adrenomyeloneuropathy (AMN) and inflammatory …

Adrenoleukodystrophy–neuroendocrine pathogenesis and redefinition of natural history

S Kemp, IC Huffnagel, GE Linthorst… - Nature Reviews …, 2016 - nature.com
X-Linked adrenoleukodystrophy (ALD) is a peroxisomal metabolic disorder with a highly
complex clinical presentation. ALD is caused by mutations in the ABCD1 gene, which leads …

X-linked adrenoleukodystrophy: pathogenesis and treatment

M Engelen, S Kemp, BT Poll-The - Current neurology and neuroscience …, 2014 - Springer
Abstract X-linked adrenoleukodystrophy (X-ALD) is a puzzling inborn error of metabolism
with a strikingly heterogeneous clinical spectrum. All patients have mutations in the ABCD1 …

General aspects and neuropathology of X‐linked adrenoleukodystrophy

I Ferrer, P Aubourg, A Pujol - Brain pathology, 2010 - Wiley Online Library
Abstract X‐adrenoleukodystrophy (X‐ALD) is a metabolic, peroxisomal disease affecting the
nervous system, adrenal cortex and testis resulting from inactivating mutations in ABCD1 …

X-linked adrenoleukodystrophy: genes, mutations, and phenotypes

KD Smith, S Kemp, LT Braiterman, JF Lu, HM Wei… - Neurochemical …, 1999 - Springer
Abstract X-linked adrenoleukodystrophy (X-ALD) is a complex and perplexing
neurodegenerative disorder. The metabolic abnormality, elevated levels of very long-chain …

[HTML][HTML] Pathophysiology of X-linked adrenoleukodystrophy

J Berger, S Forss-Petter, FS Eichler - Biochimie, 2014 - Elsevier
Currently the molecular basis for the clinical heterogeneity of X-linked
adrenoleukodystrophy (X-ALD) is poorly understood. The genetic bases for all different …

The changing face of adrenoleukodystrophy

J Zhu, F Eichler, A Biffi, CN Duncan… - Endocrine …, 2020 - academic.oup.com
Adrenoleukodystrophy (ALD) is a rare X-linked disorder of peroxisomal oxidation due to
mutations in ABCD1. It is a progressive condition with a variable clinical spectrum that …

Biochemical aspects of X‐linked adrenoleukodystrophy

S Kemp, R Wanders - Brain Pathology, 2010 - Wiley Online Library
Abstract X‐linked adrenoleukodystrophy (X‐ALD) is the most common peroxisomal disorder.
The disease is characterized by the accumulation of very long‐chain fatty acids (VLCFA;> …

[HTML][HTML] X-linked adrenoleukodystrophy: clinical, biochemical and pathogenetic aspects

J Berger, J Gärtner - Biochimica et Biophysica Acta (BBA)-Molecular Cell …, 2006 - Elsevier
X-linked adrenoleukodystrophy (X-ALD) is a clinically heterogeneous disorder ranging from
the severe childhood cerebral form to asymptomatic persons. The overall incidence is 1 …