A race against time
Published 3:58 pm Monday, March 24, 2014
Griffin family seeks signatures on petition for Duchenne treatment
By KATIE MCDOWELL/General Manager
Although he looks healthy, climbing steps or getting into a car can be a struggle for 8-year-old Gabe Griffin.
It hasn’t always been that way. Gabe, who lives along U.S. 280 in Shelby County, has Duchenne Muscular Dystrophy, which causes muscles to weaken over time. Duchenne is rare – It affects about one out every 3,500 boys – but it is also the leading genetic killer of children. A child diagnosed with Duchenne will likely be in a wheelchair by age 12 and will die from the disease in his late teens or early 20s.
“The disease is progressing. That’s why time is so critical,” Gabe’s father, Scott Griffin, said.
Now, the Hope for Gabe foundation – founded by Scott and his wife, Traci, to support their son and Duchenne research – has joined The Duchenne Alliance, a partnership of smaller Duchenne foundations from across the country.
“We believe there’s strength in numbers,” Scott said.
The alliance’s goal is to advance therapies that treat the disease. Specifically, the group is looking to garner 100,00 signatures on a White House petition through its “The Race to Yes” campaign, which seeks to accelerate FDA approval of a Duchenne treatment that supporters say has been effective and safe in clinical trials.
The drug is called eteplirsen, and Griffin said it has been endorsed by prominent Duchenne researchers. The drug is intended to stop the progression of the disease, not reverse it.
“For example, if you’re already in a wheelchair, it’s not going to make you walk again,” he said.
Griffin said the group has been pushing for the drug’s approval for about 18 months. They would like the FDA to approve the drug through the FDA Safety and Innovation Act, which allows the agency to “speed patient access to safe and effective products.”
“This drug was originally intended to stop the progression of the disease, which it has shown to do based on the two-year data from the trials,” Griffin said in a separate email interview. “The drug has now shown improved pulmonary function, an increase in the production of dystrophin (the protein these boys are missing), and some of the children are actually improving with zero side effects.”
Currently, there is no cure for Duchenne or any FDA-approved treatments. Many doctors recommend steroids, but they can cause serious side effects, according to a Race to Yes press release.
The Griffins are spreading the word about the Race to Yes campaign and the promise eteplirsen seems to hold. Meanwhile, Gabe is rapidly approaching his ninth birthday in May.
“It’s a shame, but we don’t really like to celebrate birthdays anymore,” Scott said.