Marshfield family hopes for FDA approval of son's medication
The Patriot Ledger
After a nine-year worldwide experimental study of Ataluren, a drug that is sold only in the
Joey has been taking Ataluren since the study began in 2008, and his family says his life wouldn't be the same without it.
"We're lucky to even have had access to this for the last nine years," Joey's dad, Mike, said. "I don't know too many kids (with Duchenne) who can stand and pivot at the age of 16. I see parents carrying kids at this age who need much more assistance, but he can make it through because of this drug."
Duchenne muscular dystrophy is a genetic disorder that causes progressive muscle degeneration and weakness. Most patients lose the ability to walk in their pre-teens, and the average life expectancy is 26.
Joey has experienced the expected symptoms of his disease. He is mostly wheelchair-bound, and he gets tired more easily than other kids his age. On the other hand, he is able to get himself in and out of his wheelchair, has above-average upper-body strength and attends
"We get together with other families and they just can't believe how strong he is at this stage," Joey's mother, Denise, said. "It's all the Ataluren."
Experimental studies of the drug started in 2008, then stopped for a year before resuming in 2011. Because the medication hasn't been approved in America, only study participants can access it. Joey takes five doses a day.
"Immediately when he went off the drug he was falling consistently,"
The Chorzewskis went to
Joey was diagnosed with Duchenne when he was 4 years old. At
"It makes me stronger and I have more stamina," Joey said of Ataluren. "I don't need as much assistance (when I take it)."
This isn't the first time a
The family of
The two boys have different types of the rare disease and cannot take the same medications.
(c)2017 The Patriot Ledger, Quincy, Mass.
Visit The Patriot Ledger, Quincy, Mass. at www.patriotledger.com
Distributed by Tribune Content Agency, LLC.