May. 11, 2016
Posted by Christina Jensen
Debra Miller co-founded CureDuchenne in 2003 with her husband, Paul, after their only son was diagnosed with Duchenne Muscular Dystrophy. With a background in sales and marketing, she leads CureDuchenne and has funded seven research projects that have advanced to human clinical trials, three of which are the closest to becoming the first approved treatments for the disorder.
Flash back 15 years and Debra remembers living an ideal life with her husband and their beautiful, four-year-old son, Hawken. The parents enjoyed precious moments as a new family, just watching their son play – “a blue sky life” as Miller describes it.
“My son’s diagnosis was the worst day of my life,” she recalls. “It took two years of misdiagnosis and our pediatrician not taking it seriously that something wasn’t quite right with my son.”
She first heard the word Duchenne a few nights before the results of Hawken’s blood test. “It is horrendous thinking you have this perfect little child… I can describe it like losing someone, losing the future you thought you had. Everything changed.”
Hawken’s diagnosis lit a fire in Miller and her husband. In only a few years, they have paved the way for a brighter future and made a tremendous impact for families with Duchenne, a genetic disorder characterized by progressive muscle degeneration and weakness that affects 1 in 3,500 boys, claiming their lives by their mid-20s. For the first time, there is hope to find a cure for this destructive disease affecting thousands of boys and their families.
“It’s not about bake sales and running marathons,” she says. “You have to think big.”
Miller runs CureDuchenne based on a venture philanthropy model, a model for other organizations to follow. To date, she has leveraged more than $100 million in pharma and biotech research and development investments.
Recently she launched a new program, CureDuchenne Cares, to train physical therapists around the country. “There is a huge need for a physical therapy standard of care and not many experienced Duchenne centers exist around the country,” says Miller. “Many parents don’t attend national conferences… This is a way to help the patient community and we are really proud of this program.”
It is no longer only Hawken, but other families in the Duchenne community that keep her motivated. She says, “I only have one child, but I have grown a heart for all these kids.”
NORD is honored to share Debra’s story and to honor her with a Rare Impact Award. Read the stories of all of NORD’s
2016 Rare Impact Award Honorees.