IT CAN KILL KIDS BEFORE THEIR FIRST BIRTHDAY.
THE INFANTILE FORM OF POMPE DISEASE IS A DEATH SENTENCE IF NOT TREATED.
NOW THERE'S SOMETHING KEEPING KIDS ALIVE LONGER.
IF HALEY HAYES' PARENTS COULD HAVE ONE WISH, IT WOULD BE A CURE FOR THEIR SIX-YEAR-OLD DAUGHTER. HALEY WAS DIAGNOSED WITH POMPE DISEASE, A FATAL, GENETIC ILLNESS WHEN SHE WAS SIX MONTHS OLD.
"It was awful. That was probably just the worst news you could ever hear."
HALEY LACKS AN ENZYME THAT HELPS BREAK DOWN SUGAR. SO IT BUILDS UP IN HER HEART AND SKELETAL MUSCLES, DAMAGING THEM. WHEN HALEY WAS DIAGNOSED - MOST BABIES WITH POMPE DISEASE DIED.
BUT, THIS ONCE-A-WEEK INFUSION GIVEN WHILE HALEY PAINTS, HAS CHANGED EVERYTHING.
"We know it's basically keeping her alive at this moment."
MYOZYME REPLACES THE ENZYME HALEY LACKS. WITHOUT IT, MOST BABIES DIDN'T MAKE IT TO THEIR FIRST BIRTHDAY.
"It's similar to providing insulin to someone who has diabetes, so she needs to have this enzyme given to her throughout her life course to be able to live."
BUT SOME PATIENTS CREATE ANTIBODIES THAT PREVENT THEM FROM RESPONDING TO MYOZYME.
DUKE DOCTORS FOUND THREE CHEMOTHERAPY DRUGS THAT WIPED OUT THE HARMFUL ANTIBODIES IN INFANTS, ALLOWING THEM TO RESPOND TO MYOZYME. THESE BABIES WOULD HAVE DIED, BUT THE OLDEST IS NOW FIVE.
"It allows children to live. It allows them to have dreams."
HALEY DOESN'T LET IT SLOW HER DOWN. HER PARENTS CLING TIGHT TO HOPES FOR A CURE AND ENJOY EACH PRECIOUS DAY. "Just treasure every milestone."
A LITTLE GIRL WHO REMINDS THEM OF THAT WITH EVERY SMILE.
WHILE HALEY TAKES MYOZYME ONCE A WEEK MOST PATIENTS TAKE IT ONCE EVERY TWO WEEKS.
MYOZYME IS NOT A CURE.
SINCE IT'S SO NEW DOCTORS DON'T YET KNOW HOW LONG KIDS TREATED WITH IT WILL LIVE.
THE OLDEST LIVING CHILD TAKING IT IS 13 YEARS OLD. DOCTORS SAY IT'S VITAL TO START CHILDREN WITH MYOZYME AS SOON AS POSSIBLE.
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