Two Vermont brothers have the same debilitating condition — Duchenne muscular dystrophy. Max Leclaire, 10, is on an experimental drug called eteplirsen, which is visibly helping him with his condition by making him stronger.
Max’s 13-year-old brother, Austin, on the other hand, is not on the drug, and his condition is getting worse.
That’s because Austin did not meet the qualifications to be part of the same drug trial to receive eteplirsen as Max, partly because he is not able to walk, WCAX reported.
“I’m jealous of him, but I’m happy for him, too,” Austin told the Brattleboro Reformer. “If I lose more of my strength I might not be able to hold myself up.”
The brothers’ parents are asking the drug company, Sarepta Therapeutics, to provide the same drug to Austin, too, now that they see it has helped their other son. But their requests have yet to be approved.
“I am clearly happy for Max because he is doing so well and exceeding our expectations for this drug,” Jenn Mcnary, the brothers’ mother, told ABC News. “But it’s taking much longer than we ever thought for the drug company to at least grant access to his brother [Austin] who is sitting home watching his brother get treatment.”
Sarepta Therapeutics told ABC News that while the company is sympathetic to Austin’s case, it’s a “challenging request since it involves complex regulatory, political, manufacturing, and fiscal issues that need to be addressed before a compassionate access program can be developed.”
The Brattleboro Reformer reported that the drug works by helping the body to produce a protein called dystrophin that is lacked by people who have Duchenne muscular dystrophy.
The condition only affects boys, though females can be carriers for the disease, according to the A.D.A.M. Medical Encyclopedia. It occurs in one of every 3,600 male births.
People with the condition gradually lose their ability to walk, and will eventually begin to have trouble breathing when they reach age 20, the A.D.A.M. Medical Encyclopedia reported.