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"Lorenzo's oil" therapy for X-linked adrenoleukodystrophy: rationale and current assessment of efficacy.
[adrenomyeloneuropathy]
X-
linked
adrenoleukodystrophy
(
X-
ALD
)
is
a
genetic
disorder
that
damages
the
nervous
system
and
is
associated
with
the
accumulation
of
saturated
very
long
chain
fatty
acids
(
SVLCFA
)
.
Oral
administration
of
"
Lorenzo
's
oil
"
(
LO
)
,
a
4
:
1
mixture
of
glyceryl
trioleate
and
glyceryl
trierucate
,
normalizes
the
SVLCFA
levels
in
plasma
,
but
its
clinical
efficacy
and
the
clinical
indications
for
its
use
have
been
controversial
for
more
than
15
years
.
We
review
the
biochemical
effects
of
LO
administration
and
the
rationale
for
its
use
and
present
a
current
appraisal
of
its
capacity
to
reduce
the
risk
for
the
childhood
cerebral
phenotype
when
administered
to
asymptomatic
boys
and
to
slow
progression
of
adrenomyeloneuropathy
in
patients
without
cerebral
involvement
.
We
also
present
current
efforts
to
provide
definitive
evaluation
of
its
clinical
efficacy
and
discuss
its
possible
role
in
the
new
therapeutic
opportunities
that
will
arise
if
newborn
screening
for
X-
ALD
is
validated
and
implemented
.
Diseases
Validation
Diseases presenting
"the accumulation of saturated very long chain fatty acids"
symptom
adrenomyeloneuropathy
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