Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy
AO Gupta, G Raymond, EI Pierpont… - Expert opinion on …, 2022 - Taylor & Francis
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 …
incidence of 1 in 14–17,000 male births, caused by pathogenic variants within the ABCD1 …
Adult-onset genetic central nervous system disorders masquerading as acquired neuroinflammatory disorders: a review
X Ayrignac, C Carra-Dallière, C Marelli, G Taïeb… - JAMA …, 2022 - jamanetwork.com
Importance Adult-onset genetic disorders may present with clinical and magnetic resonance
imaging (MRI) features suggestive of acquired inflammatory diseases. An ever-growing …
imaging (MRI) features suggestive of acquired inflammatory diseases. An ever-growing …
[HTML][HTML] X-linked adrenoleukodystrophy
X-linked adrenoleukodystrophy (X-ALD) affects the nervous system white matter and the
adrenal cortex. Three main phenotypes are seen in affected males: The childhood cerebral …
adrenal cortex. Three main phenotypes are seen in affected males: The childhood cerebral …
Presymptomatic lesion in childhood cerebral adrenoleukodystrophy: timing and treatment
EJ Mallack, KP Van Haren, A Torrey, S Van De Stadt… - Neurology, 2022 - AAN Enterprises
Background and Objectives We sought to characterize the natural history and standard-of-
care practices between the radiologic appearance of brain lesions, the appearance of …
care practices between the radiologic appearance of brain lesions, the appearance of …
[HTML][HTML] X-Linked Adrenoleukodystrophy Synonym: X-ALD
X-linked adrenoleukodystrophy (X-ALD) involves the central or peripheral nervous system
and the adrenal cortex. The nervous system and adrenal glands are involved independently; …
and the adrenal cortex. The nervous system and adrenal glands are involved independently; …
Zerebrale Adrenoleuko-dystrophie: Wachstums-verhalten früher Läsionen
E Mallack - thieme-connect.com
Ursache der Störung ist eine Mutation im ABCD1-Gen, die dazu führt, dass sehr langkettige
Fettsäuren nicht mehr abgebaut werden können und im Gewebe abgelagert werden. In den …
Fettsäuren nicht mehr abgebaut werden können und im Gewebe abgelagert werden. In den …
