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Wiskott-Aldrich syndrome: diagnosis, current management, and emerging treatments.
[wiskott-aldrich syndrome]
Wiskott-
Aldrich
syndrome
(
WAS
)
is
a
rare
X-
linked
primary
immunodeficiency
disorder
characterized
by
the
triad
of
eczema
,
thrombocytopenia
,
and
severe
and
often
recurrent
infections
.
Despite
the
rarity
of
this
disorder
,
our
understanding
of
the
molecular
and
cellular
pathogenesis
of
WAS
has
continued
to
increase
.
Advances
in
the
use
of
diagnostic
tools
,
the
provision
of
supportive
care
,
and
improvements
in
allogeneic
hematopoietic
stem
cell
transplantation
have
significantly
reduced
the
morbidity
and
mortality
associated
with
this
disorder
.
Exciting
advancements
in
the
care
of
patients
with
WAS
have
also
occurred
,
including
the
successful
application
of
autologous
gene
-modified
hematopoietic
stem
cell
transplantation
.
Diseases
Validation
Diseases presenting
"often recurrent infections"
symptom
wiskott-aldrich syndrome
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