Today, as I sit here writing this in Berkeley, California, my son Cory sits in a classroom in Missoula, Montana, learning math, English, science, history and other skills to help him through the life I hope he has. Inside him, his muscles are gradually deteriorating because of a genetic flaw in his DNA that keeps his body from producing dystrophin – a vital protein that nourishes muscles.
Meanwhile, in New Orleans, Louisiana, a doctor neither me nor Cory have ever heard of or ever met, named Jerry Mendell, from Nationwide Children’s Hospital in Columbus, Ohio, is giving a presentation at the American Academy of Neurology’s 64th Annual Meeting with the daunting title: “A Phase IIb Pacebo-Controlled Study of the Exon-Skipping Drug Eteplirsen in Subjects with Duchenne Muscular Dystrophy.”
As smart as Cory is, I am certain he wouldn’t care about or comprehend what Dr. Mendell is talking about. I sure don’t grasp much of it. But here’s the part I do understand: The doctor is discussing results of a recent, 24-week long clinical trial, using a drug called Eteplirsen, that significantly increased the presence of dystrophin in boys with Duchenne.
In other words, simply translated: HOPE!
And in Hackensack, New Jersey, a determined, dedicated group of people who work for Parent Project Muscular Dystrophy (of which the founder lost two sons to Duchenne) is tirelessly striving away to raise awareness and money to help fund critical, promising research like the study Dr. Mendell is presenting today.
Berkeley, Missoula, New Orleans, Columbus, Hackensack . . . all over the nation, all over the world, there are boys and young men with Duchenne, researchers working on potential treatments and a cure, and concerned people helping in any and every way they can. I am feeling good about all these connections, and confident it will all come together someday soon.
I hope you will add your name and place to these connections and help us turn hope into reality.
Please check out my other blog posts at: The Cory Story.com