A Napa Valley fundraiser in Newport Beach, California, earlier this month attracted more than 400 people and raised at least $1 million for Duchenne muscular dystrophy (DMD) research.
Among the event’s important guests were the deLeuze family of ZD Wines, honorary chair of the event, and chef Amar Santana, owner of two California restaurants: Broadway by Amar Santana in Laguna Beach and Vaca in Costa Mesa. Santana prepared the event’s meal — specially conceived for the occasion — and the auction featured a private dinner with the chef.
Guests heard first-hand what it’s like to live with DMD when 20-year-old Hawken Miller took the floor.
“Just 10 years ago, I could play handball with my friends. Now I’m afraid that if I walk too far I might fall,” Hawken said at the event, according to a press release. “Simple things, like getting out of a chair, making my bed, getting dressed and writing have gotten harder and harder. I fall more frequently, and that’s one of the hardest parts of the disease. It makes me feel helpless. Being 20 years old with Duchenne is scary. I’m not supposed to have lived this long. The worst part is I don’t know how long I have left.”
Another DMD patient, Jack Wolf, joined Miller on stage to show the audience a video featuring himself in a CureDuchenne campaign.
Miller’s mother, Debra Miller, is CureDuchenne’s founder and CEO. She told her audience how her organization provided early funding for Exondys 51 (eteplirsen), the first approved treatment designed to target the underlying cause of DMD. The U.S. Food and Drug Administration approved Exondys 51 in September 2016.
“Our successes over the past year remind us how much we can accomplish together,” Miller said. “With the first treatments available, we are one step closer to our mission of finding a cure for Duchenne, but we have a long way to go.”
All of the event’s proceeds directly fund CRISPR/Cas9 technology research, a one-time treatment the organization says could permanently correct the mutation that causes DMD. A rare genetic disorder characterized by progressive muscle deterioration and weakness, DMD occurs in about one out of every 3,600 male infants worldwide.