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Treatment of sporadic inclusion body myositis with bimagrumab.
[inclusion body myositis]
To
study
activin
signaling
and
its
blockade
in
sporadic
inclusion
body
myositis
(
sIBM
)
through
translational
studies
and
a
randomized
controlled
trial
.
We
measured
transforming
growth
factor
β
signaling
by
SMAD
2
/
3
phosphorylation
in
muscle
biopsies
of
50
patients
with
neuromuscular
disease
(
17
with
sIBM
)
.
We
tested
inhibition
of
activin
receptors
IIA
and
IIB
(
ActRII
)
in
14
patients
with
sIBM
using
one
dose
of
bimagrumab
(
n
=
11
)
or
placebo
(
n
=
3
)
.
The
primary
outcome
was
the
change
in
right
thigh
muscle
volume
by
MRI
at
8
weeks
.
Lean
body
mass
,
strength
,
and
function
were
secondary
outcomes
.
Twelve
of
the
patients
(
10
bimagrumab
,
2
placebo
)
participated
in
a
subsequent
16
-
week
observation
phase
.
Muscle
SMAD
2
/
3
phosphorylation
was
higher
in
sIBM
than
in
other
muscle
diseases
studied
(
p
=
0
.
003
)
.
Eight
weeks
after
dosing
,
the
bimagrumab-treated
patients
increased
thigh
muscle
volume
(
right
leg
+
6
.
5
%
compared
with
placebo
,
p
=
0
.
024
;
left
leg
+
7
.
6
%
,
p
=
0
.
009
)
and
lean
body
mass
(
+
5
.
7
%
compared
with
placebo
,
p
=
0
.
014
)
.
Subsequently
,
bimagrumab-treated
patients
had
improved
6
-
minute
walking
distance
,
which
peaked
at
16
weeks
(
+
14
.
6
%
,
p
=
0
.
008
)
compared
with
placebo
.
There
were
no
serious
adverse
events
;
the
main
adverse
events
with
bimagrumab
were
mild
acne
and
transient
involuntary
muscle
contractions
.
Transforming
growth
factor
β
superfamily
signaling
,
at
least
through
ActRII
,
is
implicated
in
the
pathophysiology
of
sIBM
.
Inhibition
of
ActRII
increased
muscle
mass
and
function
in
this
pilot
trial
,
offering
a
potential
novel
treatment
of
sIBM
.
This
study
provides
Class
I
evidence
that
for
patients
with
inclusion
body
myositis
,
bimagrumab
increases
thigh
muscle
volume
at
8
weeks
.
Diseases
Validation
Diseases presenting
"secondary outcomes"
symptom
acute rheumatic fever
congenital diaphragmatic hernia
heparin-induced thrombocytopenia
hydrocephalus with stenosis of the aqueduct of sylvius
inclusion body myositis
thoracic outlet syndrome
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