Vermont Brothers Get Deadly Disease, Only One Gets Treatment
Max and Austen LeClaire, from Vermont, were both born with Duchenne Muscular Dystrophy, but only Max has been approved for a new clinical treatment.
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.
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.
What is Duchenne Muscular Dystrophe?
Duchenne muscular dystrophy is an inherited disorder that involves muscle weakness, which quickly gets worse.
Other muscular dystrophies (including Becker's muscular dystrophy) get worse much more slowly. Because of the way the disease is inherited, boys are affected, not girls. The sons of females who are carriers of the disease (women with a defective gene but no symptoms themselves) each have a 50% chance of having the disease. The daughters each have a 50% chance of being carriers.
Duchenne muscular dystrophy occurs in about 1 out of every 3,600 male infants. Because this is an inherited disorder, risks include a family history of Duchenne muscular dystrophy.