Rare Disease Report

A Day in the Life of a Duchenne Parent

APRIL 17, 2017
James Raffone, JAR of Hope

James Raffone, Founder and CEO of JAR of Hope talks about the struggles in raising a boy with Duchenne muscular dystrophy.

As James explains in the video above, it is heartbreaking as a parent to watch your son slowly deteriorate while be frustrated by the lack of treatment options available. 

Continuing the conversation in the video clip below, James believes his son's future is bleak unless something is done now to change the trajectory of this devasting disease.

For more information about the JAR of Hope, visit jarofhope.org

What is Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy is a progressive muscle disorder caused by the lack of functional dystrophin protein. Patients with Duchenne muscular dystrophy lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties.

There are an estimated 35,000 patients with DMD in the United States and Europe but the population has many subsets based on mutations of the dystrophin gene.

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