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Amiloride as an Alternate Adjuvant Antiproteinuric Agent in Fabry Disease: The Potential Roles of Plasmin and uPAR.
[fabry disease]
Patients
with
Fabry
disease
present
a
higher
risk
of
cardiovascular
and
kidney
morbidity
.
We
present
a
patient
with
a
past
history
of
biopsy-proven
Fabry
disease
and
stage
3
chronic
kidney
disease
.
Proteinuria
partially
dropped
from
6
.
8
g
/
day
to
2
.
1
g
/
day
despite
an
aggressive
regime
which
consisted
of
low
-
salt
diet
,
agalsidase
beta
infusions
,
dual
blockade
of
the
renin
-angiotensin
system
,
and
low
-dose
maintenance
of
steroids
.
As
proteinuria
is
considered
a
risk
marker
of
cardiovascular
disease
and
of
progression
of
kidney
disease
,
we
added
amiloride
5
mg
/
day
,
a
drug
with
proven
effects
in
podocyte
stabilization
and
proteinuria
actions
at
the
distal
convoluted
tubule
.
Proteinuria
finally
decreased
to
0
.
8
g
/
day
.
This
report
highlights
the
relevance
of
intervening
on
proteinuria
in
a
multitarget
approach
in
order
to
reduce
it
as
much
as
possible
.
Due
to
this
pharmacological
response
,
we
suggest
that
although
agalsidase
beta
specific
treatment
protects
the
endothelium
,
the
podocyte
,
and
the
tubule
in
Fabry
disease
and
secondary
haemodynamic
and
immunologic
pathways
are
treated
with
inhibition
of
the
renin
-angiotensin
system
and
steroids
,
amiloride
may
act
as
a
complementary
tool
in
podocyte
stabilization
and
in
proteinuria
effects
at
the
distal
tubule
.
Diseases
Validation
Diseases presenting
"proven effects in podocyte stabilization and proteinuria actions at the distal"
symptom
fabry disease
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