For the past decade, around the world, the last day in February has been observed as “Rare Disease Day.”
It’s a time families bring light to rare illnesses plaguing their loved ones.
KRON4’s Philippe Djegal met with a Bay Area family fighting to keep their young daughter alive.
“I think we can beat it,” Doug Dooley said. “I think we’re going to get it for her.”
Doug and his wife Christine Waggoner do their best to put on a brave face for their 10-year-old daughter Iris.
But some days are easier than others.
“We try to enjoy as much as we can and not try to let the shadow, you know, you try and not notice the shadow that’s kind of over your life and just enjoy the moment you have,” Dooley said.
Doug and Christine shared Iris’ story in San Francisco in between doctor’s appointments at UCSF Benioff Children’s hospital.
Four and a half years ago, Iris was diagnosed with the rare and deadly degenerative neurological disease called GM1 gangliosidosis.
“Thousands of people are dying of this condition and the vast majority are children,” Christine said.
About three years ago, Doug and Christine started a foundation and launched a website: http://curegm1.org/
In that time, they’ve raised about two million dollars. All of it going to fund research to help find a cure but they could still use more donations.
“One in 200,000 people are born with GM1 gangliosidosis, Christine said. “And, one in 250 people carry the gene for the disease.”
The hope is to raise enough money so iris can participate in an experimental gene therapy clinical trial.
“The moments here and the technology is here to really start curing all these genetic diseases, and, you know, we just want to be apart and help that happen so that families don’t have to suffer.”
And, on Rare Disease Day, the message is, help if you can.
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