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Afamelanotide, an agonistic analog of α-melanocyte-stimulating hormone, in dermal phototoxicity of erythropoietic protoporphyria.
[erythropoietic protoporphyria]
Afamelanotide
,
an
α-melanocyte
stimulating
hormone
(
MSH
)
agonistic
analog
is
a
first
-
in
-class
therapeutic
.
Its
application
to
protoporphyria
(
PP
)
,
a
disease
associated
with
absolute
sunlight-intolerance
is
discussed
.
The
genetics
and
existing
therapy
of
the
inherited
disease
PP
comprising
both
erythropoietic
protoporphyria
and
X-
linked
dominant
protoporphyria
.
The
physiological
and
pharmacological
actions
of
α-
MSH
and
afamelanotide
including
receptor-mediated
intracellular
signaling
and
effects
of
receptor
polymorphisms
.
Adverse
effects
and
safety
issues
.
The
clinical
severity
and
the
necessity
for
an
effective
therapy
for
the
rare
disease
PP
are
illustrated
by
a
short
,
up-
to
-date
portrait
.
A
condensed
description
of
clinically
important
aspects
of
α-
MSH
signaling
,
physiological
,
pharmacological
and
safety
issues
of
afamelanotide
applied
to
humans
and
the
rational
for
its
potential
efficacy
in
PP
are
given
.
The
different
trials
of
afamelanotide
in
PP
and
their
most
recent
results
are
discussed
.
Although
early
,
results
of
the
first
trials
of
afamelanotide
for
PP
are
promising
and
the
risk-safety
profile
appears
favorable
today
.
We
expect
afamelanotide
and
analogs
thereof
to
be
a
prospective
therapeutic
tool
in
light-related
skin
diseases
,
and
in
future
this
drug
class
might
prove
effectiveness
in
other
medical
conditions
.
Diseases
Validation
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"and in future this drug class might prove effectiveness in other medical conditions"
symptom
erythropoietic protoporphyria
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