Within the Duchenne muscular dystrophy community, I’ve earned membership in what we call the “old mom club.” My son is 33 years old—an age many boys with Duchenne previously did not have a chance to reach. 

My son’s life is a testament to scientific progress. A college graduate, one reason he has lived so long is that his genetic mutation responded well to a treatment designed for his specific subset of Duchenne. This treatment helped preserve functions that would have previously been lost much earlier. 

Duchenne has been a learning curve for all of us, but it has taught me a great lesson: There is no such thing as a single miracle treatment. 

Today, while my son’s pulmonary function remains stable, his cardiac health is declining. That reflects the complexity of Duchenne, as its dystrophin mutations affect patients differently and progress along different paths, organ systems, and timelines. 

Since 2011, Jon has been on the therapeutic Atalauren—the first genetic treatment reviewed by the FDA to target an underlying cause of Duchenne. Although it has not yet been approved by the FDA, he has continued the treatment under the agency’s “extended access,” because he was in the original trial. 

It has........

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