European Journal of Pediatrics,
Journal Year:
2024,
Volume and Issue:
183(9), P. 4057 - 4062
Published: July 3, 2024
Abstract
Duchenne
muscular
dystrophy
is
life-limiting.
Cardiomyopathy,
which
mostly
ensues
in
the
second
decade
of
life,
main
cause
death.
Treatment
options
are
still
limited.
The
TAMDMD
(NCT03354039)
trial
assessed
motor
function,
muscle
strength
and
structure,
laboratory
biomarkers,
safety
79
ambulant
boys
with
genetically
confirmed
dystrophy,
6.5–12
years
age,
receiving
either
daily
tamoxifen
20
mg
or
placebo
for
48
weeks.
In
this
post-hoc
analysis,
available
echocardiographic
data
patients
recruited
at
one
study
centre
were
retrieved
compared
before
after
treatment.
Data
from
14
patients,
median
11
(interquartile
range,
IQR,
11–12)
age
was
available.
Baseline
demographic
characteristics
similar
participants
assigned
to
(
n
=
7)
7).
Left
ventricular
end-diastolic
diameter
group
(median
IQR)
39
(38–41)
mm
baseline
43
(38–44)
end,
while
it
44
(41–46)
41
(37–46)
treatment
group.
fractional
shortening
35%
(32–38%)
33%
(32–36%)
treatment,
34%
(33–34%)
(33–35%)
end.
No
signals
detected.
Conclusion
:
This
hypothesis-generating
analysis
suggests
that
over
weeks
well
tolerated
may
help
preserving
cardiac
structure
function
dystrophy.
Further
studies
justified.
ClinicalTrials.gov
Identifier
EudraCT
2017–004554–42,
NCT03354039
What
known:
•
(DMD)
Cardiomyopathy
life
Tamoxifen
reduced
fibrosis
mice
improved
cardiomyocyte
human-induced
pluripotent
stem
cell-derived
cardiomyocytes.
new:
among
boys,
treated
weeks,
well-tolerated.
A
visual
trend
left-ventricular
dimensions
better
systolic
preservation
generates
hypothesis
a
potential
beneficial
effect
DMD
cardiomyopathy.
Circulation Research,
Journal Year:
2023,
Volume and Issue:
132(10), P. 1374 - 1386
Published: May 11, 2023
COVID-19
is
an
infectious
disease
caused
by
SARS-CoV-2
leading
to
the
ongoing
global
pandemic.
Infected
patients
developed
a
range
of
respiratory
symptoms,
including
failure,
as
well
other
extrapulmonary
complications.
Multiple
comorbidities,
hypertension,
diabetes,
cardiovascular
diseases,
and
chronic
kidney
are
associated
with
severity
increased
mortality
COVID-19.
infection
also
causes
complications,
myocarditis,
myocardial
injury,
heart
arrhythmias,
acute
coronary
syndrome,
venous
thromboembolism.
Although
variety
methods
have
been
many
clinical
trials
launched
for
drug
repositioning
COVID-19,
treatments
that
consider
manifestations
comorbidities
specifically
limited.
In
this
review,
we
summarize
recent
advances
in
experimental
repositioning,
high-throughput
screening,
omics
data-based,
network
medicine-based
computational
particular
attention
on
those
We
discuss
prospective
opportunities
potential
repurposing
drugs
treat
complications
European Heart Journal - Cardiovascular Pharmacotherapy,
Journal Year:
2023,
Volume and Issue:
9(8), P. 758 - 769
Published: Aug. 10, 2023
Abstract
Although
bioinformatic
methods
gained
a
lot
of
attention
in
the
latest
years,
their
use
real-world
studies
for
primary
and
secondary
prevention
atherosclerotic
cardiovascular
diseases
(ASCVD)
is
still
lacking.
Bioinformatic
resources
have
been
applied
to
thousands
individuals
from
Framingham
Heart
Study
as
well
health
care-associated
biobanks
such
UK
Biobank,
Million
Veteran
Program,
CARDIoGRAMplusC4D
Consortium
randomized
controlled
trials
(i.e.
ODYSSEY,
FOURIER,
ASPREE,
PREDIMED).
These
contributed
development
polygenic
risk
scores
(PRS),
which
emerged
novel
potent
genetic-oriented
tools,
able
calculate
individual
ASCVD
predict
response
therapies
statins
proprotein
convertase
subtilisin/kexin
type
9
inhibitor.
are
first
cause
death
around
world
including
coronary
heart
disease
(CHD),
peripheral
artery
disease,
stroke.
To
achieve
goal
precision
medicine
personalized
therapy,
advanced
platforms
set
link
clinically
useful
indices
heterogeneous
molecular
data,
mainly
epigenomics,
transcriptomics,
metabolomics,
proteomics.
The
DIANA
study
found
that
differential
methylation
ABCA1,
TCF7,
PDGFA,
PRKCZ
significantly
discriminated
patients
with
acute
syndrome
healthy
subjects
expression
levels
positively
associated
CK-MB
serum
concentrations.
ARIC
revealed
several
plasma
proteins,
acting
or
not
lipid
metabolism,
potential
role
determining
different
pleiotropic
effects
each
subject.
implementation
high-throughput
techniques
into
traditional
prediction
emerging
more
accurate
practice
stratify
earlier
life
favour
timely
tailored
reduction
strategies.
Of
note,
radiogenomics
aims
combine
imaging
features
extracted
instance
by
computed
tomography
angiography
biomarkers
create
CHD
diagnostic
algorithms
characterize
lesions
myocardial
abnormalities.
current
view
could
be
clinical
value
prevention,
stratification,
treatment
ASCVD.
Current Pharmaceutical Design,
Journal Year:
2024,
Volume and Issue:
30(19), P. 1459 - 1471
Published: April 15, 2024
Neurological
disorders
impose
a
significant
burden
on
individuals,
leading
to
disabilities
and
reduced
quality
of
life.
However,
recent
years
have
witnessed
remarkable
advancements
in
pharmaceutical
interventions
aimed
at
treating
these
disorders.
This
review
article
aims
provide
an
overview
the
latest
innovations
breakthroughs
neurological
disorder
treatment,
with
specific
focus
key
therapeutic
areas
such
as
Alzheimer's
disease,
Parkinson's
multiple
sclerosis,
epilepsy,
stroke.
explores
emerging
trends
drug
development,
including
identification
novel
targets,
development
innovative
delivery
systems,
application
personalized
medicine
approaches.
Furthermore,
it
highlights
integration
advanced
technologies
gene
therapy,
optogenetics,
neurostimulation
techniques.
These
hold
promise
for
precise
modulation
neural
circuits,
restoration
neuronal
function,
even
disease
modification.
While
offer
hopeful
prospects
more
effective
tailored
treatments,
challenges
need
improved
diagnostic
tools,
new
targets
intervention,
optimization
methods
will
remain.
By
addressing
continuing
invest
research
collaboration,
we
can
revolutionize
treatment
significantly
enhance
lives
those
affected
by
conditions.
Circulation,
Journal Year:
2023,
Volume and Issue:
148(22), P. 1778 - 1796
Published: Oct. 31, 2023
Kawasaki
disease
(KD)
and
multisystem
inflammatory
syndrome
in
children
(MIS-C)
share
similar
clinical
manifestations,
including
cardiovascular
complications,
suggesting
underlying
immunopathogenic
processes.
Aberrant
neutrophil
activation
may
play
a
crucial
role
the
shared
pathologies
of
KD
MIS-C;
however,
associated
pathogenic
mechanisms
molecular
drivers
remain
unknown.
Biochemical Society Transactions,
Journal Year:
2024,
Volume and Issue:
52(3), P. 1045 - 1059
Published: May 23, 2024
Major
advancements
in
human
pluripotent
stem
cell
(hPSC)
technology
over
recent
years
have
yielded
valuable
tools
for
cardiovascular
research.
Multi-cell
type
3-dimensional
(3D)
cardiac
models
particular,
are
providing
complementary
approaches
to
animal
studies
that
better
representatives
than
simple
2-dimensional
(2D)
cultures
of
differentiated
hPSCs.
These
3D
can
be
broadly
divided
into
two
categories;
namely
those
generated
through
aggregating
pre-differentiated
cells
and
form
self-organizing
structures
during
their
vitro
differentiation
from
either
replicate
aspects
development
or
enable
the
examination
interactions
among
constituent
types,
with
some
these
showing
increased
maturity
compared
2D
systems.
Both
groups
already
emerged
as
physiologically
relevant
pre-clinical
platforms
studying
heart
disease
mechanisms,
exhibiting
key
functional
attributes
heart.
In
this
review,
we
describe
different
organoid
derived
hPSCs,
generation
methods,
applications
research
use
drug
screening.
We
also
address
current
limitations
challenges
testing
propose
potential
improvements
enhance
efficacy
discovery.
Frontiers in Cardiovascular Medicine,
Journal Year:
2024,
Volume and Issue:
11
Published: July 1, 2024
Cardiovascular
disease
(CVD)
represents
the
leading
cause
of
mortality
and
disability
all
over
world.
Identifying
new
targeted
therapeutic
approaches
has
become
a
priority
biomedical
research
to
improve
patient
outcomes
quality
life.
The
RAS-RAF-MEK
(mitogen-activated
protein
kinase
kinase)-ERK
(extracellular
signal-regulated
kinase)
pathway
is
gaining
growing
interest
as
potential
signaling
cascade
implicated
in
pathogenesis
CVD.
This
pivotal
regulating
cellular
processes
like
proliferation,
growth,
migration,
differentiation,
survival,
which
are
vital
maintaining
cardiovascular
homeostasis.
In
addition,
ERK
involved
controlling
angiogenesis,
vascular
tone,
myocardial
contractility,
oxidative
stress.
Dysregulation
this
been
linked
cell
dysfunction
cardiac
pathological
remodeling,
contribute
onset
progression
Recent
ongoing
provided
insights
into
interventions
targeting
RAS-RAF-MEK-ERK
pathologies.
Preclinical
studies
have
demonstrated
efficacy
therapy
with
MEK
inhibitors
(MEKI)
attenuating
activation
mitigating
CVD
animal
models.
article,
we
first
describe
how
contributes
preserving
health.
We
then
summarize
current
knowledge
roles
played
by
development
disorders,
including
atherosclerosis,
infarction,
hypertrophy,
heart
failure,
aortic
aneurysm.
finally
report
novel
strategies
for
these
CVDs
encompassing
MEKI
discuss
advantages,
challenges,
future
developments
therapeutics.
Journal of Materials Chemistry B,
Journal Year:
2022,
Volume and Issue:
11(3), P. 546 - 559
Published: Dec. 5, 2022
Cardiovascular
disease
(CVD),
a
type
of
circulatory
system
related
to
the
lesions
cardiovascular
system,
has
become
one
main
diseases
that
endanger
human
health.
Currently,
clinical
diagnosis
most
CVDs
relies
on
combination
imaging
technology
and
blood
biochemical
test.
However,
existing
technologies
for
still
have
limitations
in
terms
specificity,
detection
range,
cost.
In
order
break
through
current
bottleneck,
microfluidic
with
advantages
low
cost,
simple
instruments
easy
integration,
been
developed
play
an
important
role
early
prevention,
treatment
CVDs.
Here,
we
reviewed
recent
various
applications
CVDs,
including
devices
detecting
CVD
markers,
models
based
microfluidic,
used
drug
screening
delivery.
addition,
briefly
looked
forward
prospects
challenges
microfluidics
Skin
cancer
represents
a
major
health
concern
due
to
its
rising
incidence
and
limited
treatment
options.
Current
treatments
(surgery,
chemotherapy,
radiotherapy,
immunotherapy,
targeted
therapy)
often
entail
high
costs,
patient
inconvenience,
significant
adverse
effects,
therapeutic
efficacy.
The
search
for
novel
options
is
also
marked
by
the
capital
investment
extensive
development
involved
in
drug
discovery
process.
In
response
these
challenges,
repurposing
existing
drugs
topical
application
optimizing
their
delivery
through
nanotechnology
could
be
answer.
This
innovative
strategy
aims
combine
advantages
of
known
pharmacological
background
commonly
used
expedite
development,
with
nanosystem-based
formulations,
which
among
other
allow
improved
skin
permeation
retention
overall
higher
efficacy
safety.
present
review
provides
critical
analysis
repurposed
such
as
doxycycline,
itraconazole,
niclosamide,
simvastatin,
leflunomide,
metformin,
celecoxib,
formulated
into
different
nanosystems,
namely,
nanoemulsions
nanoemulgels,
nanodispersions,
solid
lipid
nanoparticles,
nanostructured
carriers,
polymeric
hybrid
lipid-polymer
electrospun
nanofibrous
scaffolds,
liposomes
liposomal
gels,
ethosomes
ethosomal
aspasomes,
outcomes
battle
against
cancer.
Enhanced
antitumor
effects
on
melanoma
nonmelanoma
research
models
are
highlighted,
some
nanoparticles
even
showing
intrinsic
anticancer
properties,
leading
synergistic
effects.
explored
findings
highly
evidence
potential
approaches
complement
currently
available
strategies
hope
that
might
one
day
reach
pharmaceutical
market.