Imagine your child is diagnosed with a disease for which there is no cure, and very little hope. And if it’s one of hundreds of “orphan diseases” that don’t garner the research dollars that bigger-name conditions do, hope for a breakthrough in treatment coming in time to help your kid dwindles.
That’s what faced Debra and Paul Miller when their son, Hawken, was diagnosed in November 2002 with Duchenne, a form of muscular dystrophy that only affects boys. The prognosis the Millers received was grim. Boys are usually diagnosed by the age of five, in a wheelchair by age 12 and most don’t survive through their mid-20s.
“The first thing we thought is, ‘Jerry Lewis has this handled, doesn’t he?’” says Paul in a video about the disease produced by CureDuchenne. It’s a national nonprofit organization located in Newport Beach the couple created when they discovered how little planning and strategic thinking went into finding a cure for the disease.
Though grass-roots in origin, the nonprofit is a sophisticated national endeavor focused on identifying and underwriting scientific research; discovering and developing treatments quickly; and using the most cutting-edge science and technology such as gene therapy and stem cell research to find a cure.
The foundation is similar to an angel investor, providing seed capital to scientists and others at very early stages of research, allowing them to make a quantum leap at a critical time, with early results triggering more substantial government and commercial funding for clinical trials and subsequent research stages. CureDuchenne’s $1.3 million early financing of Dutch biotech company Prosensa’s research led to a later commitment by GlaxoSmithKline for up to $650 million to help speed the clinical trials and approval of that company’s drug.
To date, seven research projects have made their way into human clinical trials with support from CureDuchenne. But there is one big difference between the Millers and other investors: The end goal isn’t profit, it’s saving their son’s life, and the lives of other boys just like him.
Funding research takes funds, and that’s another area at which the nonprofit excels, raising awareness of the disease through galas, golf tournaments and events like the recent Tinker Bell Half Marathon at Disneyland Resort that help produce the millions the sprint for a cure requires.
Another recent fundraiser was the Champions to Cure Duchenne gala February 9 at Balboa Bay Club & Resort, which honored hockey great Scott Niedermayer and wife Lisa. Dinner was by Josef Lageder of the BBC&R and guest chef Stan Frazier, a co-owner of A Restaurant and recent winner of “Chef Race” on BBC America.
The connection to races, athletes and athletic events is no accident. Paul Miller played college hockey at Minnesota, and the family knows the Niedermayers from Pegasus, where their kids went to school together.
But the race imagery is even more important when one imagines what motivates the passion and progress the Millers bring to the Duchenne community. “The cure is right around the corner,” they say. “But we’re in a race against time to save our boys.”
Join the Race
To learn more, call or go to CureDuchenne’s website. Or donate $10 to CureDuchenne instantly by texting the word SAVE to 80888.
949.872.2552 :: cureduchenne.org