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Docosahexaenoic acid therapy in peroxisomal diseases: results of a double-blind, randomized trial.
[zellweger syndrome]
Peroxisome
assembly
disorders
are
genetic
disorders
characterized
by
biochemical
abnormalities
,
including
low
docosahexaenoic
acid
(
DHA
)
.
The
objective
was
to
assess
whether
treatment
with
DHA
supplementation
would
improve
biochemical
abnormalities
,
visual
function
,
and
growth
in
affected
individuals
.
This
was
a
randomized
,
double
-blind
,
placebo-controlled
trial
conducted
at
a
single
center
.
Treatment
groups
received
supplements
of
DHA
(
100
mg
/
kg
per
day
)
.
The
primary
outcome
measures
were
the
change
from
baseline
in
the
visual
function
and
physical
growth
during
the
1
year
follow-up
period
.
Fifty
individuals
were
enrolled
and
randomized
.
Two
were
subsequently
excluded
from
study
analysis
when
it
was
determined
that
they
had
a
single
enzyme
disorder
of
peroxisomal
beta
oxidation
.
Thirty
-
four
returned
for
follow-up
.
Nine
patients
died
during
the
trial
of
their
disorder
,
and
5
others
were
lost
to
follow-up
.
DHA
supplementation
was
well
tolerated
.
There
was
no
difference
in
the
outcomes
between
the
treated
and
untreated
groups
in
biochemical
function
,
electroretinogram
,
or
growth
.
Improvements
were
seen
in
both
groups
in
certain
individuals
.
DHA
supplementation
did
not
improve
the
visual
function
or
growth
of
treated
individuals
with
peroxisome
assembly
disorders
.
This
interventional
study
provides
Class
II
evidence
that
DHA
supplementation
did
not
improve
the
visual
function
or
growth
of
treated
individuals
with
peroxisome
assembly
disorders
during
an
average
of
1
year
of
follow-up
in
patients
aged
1
to
144
months
.
Diseases
Validation
Diseases presenting
"visual function"
symptom
adrenomyeloneuropathy
aniridia
coats disease
cohen syndrome
congenital toxoplasmosis
homocystinuria without methylmalonic aciduria
oculocutaneous albinism
zellweger syndrome
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