In 2009, cerebral adrenoleukodystrophy (c-ALD) became the first brain disease to be treated
with lentiviral (LV)-based hematopoietic stem cell gene therapy with the ABCD1 gene in four …

Background In X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function
of the ALD protein. Cerebral adrenoleukodystrophy is characterized by demyelination and …

X-linked adrenoleukodystrophy (ALD) is a severe demyelinating disease of the brain caused
by a deficiency in ALD protein, an adenosine triphosphate--binding cassette (ABC) …

Abstract X-linked adrenoleukodystrophy (X-ALD) is a severe genetic demyelinating disease
caused by a deficiency in ALD protein, an adenosine triphosphate-binding cassette …

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 …

Allogeneic hematopoietic stem cell transplantation (HSCT) is the only therapeutic approach
that can arrest cerebral demyelination of X‐linked adrenoleukodystrophy (ALD) in boys and …

Adrenoleukodystrophy (ALD) is a demyelinating disease of the central nervous system that
results from a genetic deficiency of ALDP, an ABC protein involved in the transport of very …

X-linked adrenoleukodystrophy (ALD) is a severe brain demyelinating disease in boys that
is caused by a deficiency in ALD protein, an adenosine triphosphate–binding cassette …

X-linked adrenoleukodystrophy (ALD) is the most frequently seen genetic disorder involving
the myelin of the central nervous system. The cerebral form affects mainly boys between five …

Cerebral adrenoleukodystrophy (CALD) is a rapidly progressing, often fatal
neurodegenerative disease caused by mutations in the ABCD1 gene, resulting in deficiency …