Jenn McNary, a Vermont mom of six, is feeling bittersweet. She's watching one of her ill sons get healthy while the other deteriorates. Both of McNary's sons have Duchenne muscular dystrophy (DMD), but only 10-year-old Max has been able to participate in a study for a drug called eteplirsen, which is having dramatic effects.
After 60 weeks of IV infusion, Max has been getting stronger and some of the DMD effects have been reversed. McNary says that Max has been in a wheelchair all his life, but that he participated in a three-mile Halloween walk for the first time ever this year. (Watch this amazing feat in the home video video below!)
Austin, 13, wasn't able to participate in the trial, and so McNary has been pleading for the FDA to release what she and some other parents are calling a "miracle drug." Austin can no longer hold a cup, and will soon need a breathing machine. A new drug typically takes about four years to get approved, which would be too late for Austin. "He suffers, silently, as his disease progresses," says McNary.
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