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Hematopoietic stem cell transplantation and hematopoietic stem cell gene therapy in X-linked adrenoleukodystrophy.
[adrenomyeloneuropathy]
Allogeneic
hematopoietic
stem
cell
transplantation
(
HSCT
)
is
the
only
therapeutic
approach
that
can
arrest
cerebral
demyelination
of
X-
linked
adrenoleukodystrophy
(
ALD
)
in
boys
and
results
in
long
-term
in
a
good
quality
of
life
,
provided
the
procedure
is
performed
at
an
early
stage
of
disease
.
Similar
benefits
of
allogeneic
HSCT
have
been
demonstrated
in
adults
with
cerebral
ALD
.
However
,
it
is
not
yet
known
whether
allogeneic
HSCT
can
prevent
or
rescue
adrenomyeloneuropathy
.
Allogeneic
HSCT
remains
associated
with
significant
morbidity
and
mortality
risks
,
particularly
in
adults
,
and
not
all
ALD
patients
have
donors
despite
the
availability
of
cord
blood
.
The
absence
of
biological
markers
that
can
predict
the
evolutivity
of
cerebral
disease
is
a
major
limitation
to
propose
in
due
time
allogeneic
HSCT
to
ALD
patients
.
Recently
,
HSC
gene
therapy
using
lentiviral
vector
was
shown
to
have
comparable
efficacy
than
allogeneic
HSCT
in
two
boys
with
cerebral
ALD
who
had
no
Human-leukocyte-antigen
(
HLA
)
-
matched
donor
.
If
these
results
are
confirmed
in
an
extended
series
of
patients
,
HSC
gene
therapy
may
become
the
first
therapeutic
option
for
all
ALD
male
patients
who
develop
cerebral
demyelination
.
Diseases
Validation
Diseases presenting
"early stage"
symptom
adrenomyeloneuropathy
allergic bronchopulmonary aspergillosis
aromatase deficiency
cadasil
carcinoma of the gallbladder
child syndrome
cholangiocarcinoma
congenital adrenal hyperplasia
esophageal adenocarcinoma
esophageal carcinoma
esophageal squamous cell carcinoma
familial hypocalciuric hypercalcemia
familial mediterranean fever
gm1 gangliosidosis
harlequin ichthyosis
hereditary cerebral hemorrhage with amyloidosis
hodgkin lymphoma, classical
kindler syndrome
lymphangioleiomyomatosis
neonatal adrenoleukodystrophy
pyomyositis
scrub typhus
sneddon syndrome
typhoid
von hippel-lindau disease
zellweger syndrome
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