Inside the Nonprofit Aiming to Disrupt Big Pharma, One Case Study at a Time
By Rowan Walrath – October 31, 2019
Rich Horgan wants to save his brother’s life.
Now 24, Terry was diagnosed with Duchenne muscular dystrophy (DMD) when he was three years old. It was hardly a first for their family. Horgan had already lost several uncles to the same fate.
“This disease has really haunted my family,” Horgan said. “My mom’s three brothers all had it, all passed away from it at a young age. My brother has it now. And there’s really been nothing to advance technology to save these boys’ lives over the decades. That’s very frustrating.”
DMD is a progressive form of muscular dystrophy. Over the course of a person’s life—usually a male’s—DMD will cause skeletal and heart muscles to slowly atrophy, limiting patients’ ability to sit, stand and even speak. By the time a patient reaches adolescence, he will likely be using a wheelchair and will begin having heart and respiratory muscle problems. At the moment, there is no known cure. Just two medications have been approved by the U.S. Food and Drug Administration (FDA) as orphan products to treat it, and those only received that designation within the last three years.
As a child, Horgan says, he felt helpless watching his younger brother grow up differently—more slowly—than his peers did, than Horgan himself did.
“You start to think, ‘My brother’s not growing as fast. My brother’s not doing things that other boys his age are doing,’” Horgan said. “That framed my childhood a lot: dealing with feelings of powerlessness and hopelessness. This disease was coming no matter what.”