Facts about
Spinal Muscular Atrophy
Spinal
Muscular Atrophy (SMA) is the
leading genetic killer of infants
and toddlers. Each year 1,000
babies are born with SMA in the
U.S. and yet so many of us have
not even heard of this disease.
These infants simply do not get a
chance to grow up to be adults,
do not get a chance to become
famous, and do not make the
headlines when they die for the
world to hear about this deadly
disease.
The sad
news is . . . Whether
we have heard of SMA or not, this
little known disease
is afflicting an estimated 1 in
every 6,000 live births.
Worldwide, 13,000 lives are lost
every year. Over 25,000 Americans
have SMA.
The good
news is . . . The
international scientific
community agrees that of all
neuromuscular diseases, SMA is
the closest to developing a
treatment or a cure. Because a
cure is in sight, the National
Institutes of Health (NIH)
recently designated SMA as a
model for their research program
aimed to turn basic science into
actual drugs and treatments. It
is the educated expectation of
leading scientists and clinicians
worldwide that with the
appropriate funding, an effective
therapy for SMA can be achieved
in five years or less. Small
private investments in SMA
research have resulted in
tremendous breakthroughs.
SMA does
not affect sensation and
intellectual activity in
patients. It
commonly is observed that
patients with SMA are unusually
bright and sociable.
THE TYPES
OF SMA:
Type
1, or Werdnig-Hoffmann
Disease
Type
II SMA affects infants
between seven and 18
months old.
Type
III, also known as
Kugelberg-Welander
Disease,
Type
IV is the adult form of
the disease in which
symptoms tend to begin
after age 35.
For more
information please visit: CURE SMA
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