Deadly Disease Strikes Brothers, Only One Gets Drug

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.

Handout/Justin Ferland Photography

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.

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Deadly Disease Strikes Brothers, Only One Gets Drug