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Marshfield family hopes for FDA approval of son's medication

The Patriot Ledger

10-25-17

Oct. 24--MARSHFIELD -- The next 24 hours will be big for Joey Chorzewski, 16, a Duchenne muscular dystrophy patient who is eagerly awaiting a federal decision on the drug that keeps him strong, increases his stamina and allows him to live a semi-independent life.

After a nine-year worldwide experimental study of Ataluren, a drug that is sold only in the European Union, the federal Food and Drug Administration was slated to decide today whether the medication can be put on the market in the United States.

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 Marshfield High School full time.

"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," Denise Chorzewski said. "I was getting calls almost daily from his school, and he begged me to go back on it. So we fought for over a year to get the study resumed."

The Chorzewskis went to Washington, D.C., last month with the nonprofit Parent Project Muscular Dystrophy to testify before a committee about the drug. Historically, the FDA takes the committee's advice into consideration. Last month, the committee voted 10-1 to not approve the medication.

"The FDA is looking for a home run, but we're just looking for a base hit, enough to stay in the game," Mike Chorzewski said. "We will take any drug that provides any type of improvement."

Joey was diagnosed with Duchenne when he was 4 years old. At Marshfield High, he puts most of his energy into his passions: history and drama. Earlier this month, he was invited to join the National Honor Society.

"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 South Shore family has gone up against the FDA in the name of Duchenne.

The family of Jett McSherry of Pembroke, now 21, lobbied the government agency for more than a decade before getting the drug eteplirsen approved last year. McSherry is attending Bridgewater State University.

The two boys have different types of the rare disease and cannot take the same medications.

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