Vermont Brothers Get Deadly Disease, But Only One Gets Drug

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The Leclaire brothers were born with the same deadly disease -- Duchenne muscular dystrophy. Max, 10, is in a clinical trial for a new drug that has miraculously reversed some of his debilitating symptoms, but Austin, 13, has been turned away.

Austin watches from his wheelchair as his younger brother's strength continues to return. Max was able to climb a big sledding hill in his Saxtons River, Vt., backyard, but his older brother's muscles are so weak, he can barely feed himself.

The boys have the same gene mutation that the drug targets and will eventually kill them, but because Austin can no longer walk, he did not qualify for the trial.

"My brother says he's doing it for me, that he's trying really hard," Austin told ABCNews.com. "That's why he wanted to do it."

For the last 52 weeks, Max has been enrolled in a clinical trial with the drug eteplirsen, manufactured by Sarepta Therapeutics. Once a week, he receives intravenous infusions in a double blind study.

But after only 16 weeks, his parents suddenly realized he was on the drug and not a placebo. Not only was the progression of the disease slowing down, "he has even gained skills," said his mother, Jenn Mcnary, 32.

There is no cure for Duchenne muscular dystrophy. Until now, doctors can only use steroids, which just temporarily delay the inevitable loss of muscle strength.

"My husband noticed first at the airport," said Mcnary. "He said, 'I think he's on the drug and a high dose.' Max opened one of the McDonald's milk jugs with the sealed top. He never had that sort of grip strength."

For the last year, Max has flown to Nationwide Children's Hospital in Columbus, Ohio, for his IV treatment, but now, he goes to the Dartmouth Medical Center in nearby New Hampshire.

After seeing the success of the drug, the first of its kind, researchers put all 12 of the study subjects on that treatment.

According to Mcnary, the children on the highest dose of the drug have made a 68-meter gain in distance in six-minute walking tests.

But in the time that Max has improved, Austin has deteriorated and now can't even pick up a drinking glass, said his mother.

"He has lost his ability to unlock a door handle and leave the house and move from the bed to the wheelchair," said Mcnary. "And in the next six months, we expect more sleep apnea that will require a breathing machine."

Austin enjoys his computer, but he can no longer use his go-cart because he has lost the back strength to sit up. He is homeschooled by his mother because he gets too fatigued in a regular classroom.

So far, the Mcnarys' pleas to the drug company have fallen on deaf ears.

"I have been getting very vague answers," said Mcnary. "I keep getting the same answer. They tell me to be patient."

Dr. Edward M. Kaye, chief medical officer and senior vice president of Sarepta, wrote in an email to Mcnary that she shared with ABCNews.com that he is "sympathetic" to their case.

"This remains a priority for us to get the drug not only to Austin but to other children who may benefit from the therapy," he said in the email. "This is 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."

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