Jenn McNary's son was diagnosed with a rare form of muscular dystrophy eleven years ago. Her other son was also diagnosed with Duchenne muscular dystrophy and doctors told her there was nothing they could to about it. Duchenne muscular dystrophy is a muscle-wasting disease that afflicts roughly one in 3,600 boys. The neurologist told her it was progressive, fatal and unstoppable.
McNary was impassioned. Her younger son was granted acceptance to a clinical trial for eteplirsen - a drug that has mother has since been convinced is making him better. McNary claims that though Duchenne is a progressive disease, the kids on eteplirsen seem to be getting worse more slowly than one would expect.
“What I didn’t know was what the heartbreak of watching one child begin to recover and one child steadily decline was going to feel like in our family,” McNary says. “I became increasingly aware that I could possibly be the mother of the last child to die from Duchenne and the first child to survive it. That was unacceptable to me.”
Now, McNary among other advocates are publicly lobbying the Food and Drug Administration to grant accelerated approval for eteplirsen after Sarepta Therapeutics applies next year. Watch the video and read the full story