PHILADELPHIA--(BUSINESS WIRE)--On Saturday, April 30th, CureDuchenne, a national nonprofit that raises awareness and funds for research to find a cure for Duchenne Muscular Dystrophy, hosted a “blinged-out” event at The Logan Hotel—featuring special bingo callers including FOX29 News General Assignment Reporter, Jennaphr Frederick, former Governor Edward G. Rendell, WMMR Preston and Steve show producer, Marisa Magnatta, local sensation John Bolaris, and comedian Joe Conklin in celebration for a cause to help save the lives of those with Duchenne.
The event brought together hundreds of Philadelphia VIP’s, celebrities and influencers, raising more than $200,000. The event included a four-round game of bingo, an auction with prizes ranging from a four-night stay anywhere in the world to a luxury African safari and a pledge where guests at the event donated as much as they wanted to help find a cure for this fatal disease. Guests enjoyed music from DJ Nigel Richards along with some crafted cocktails and delicious small plates.
This year, BLINGO to CureDuchenne honored Sarepta Therapeutics, a biopharmaceutical company focused on developing innovative RNA-targeted therapeutics, as a supporter in fighting Duchenne muscular dystrophy. CureDuchenne pushes toward innovation and advancement of Duchenne research. CureDuchenne provided early funding to Sarepta which helped the company move into human clinical trials. The U.S. Food and Drug Administration (FDA) is reviewing Sarepta’s New Drug Application (NDA) for eteplirsen, an experimental drug currently in clinical trials. The FDA PDUFA date scheduled for completion of the review is May 26, 2016.
Paul Miller, co-founder of CureDuchenne presented Ed Kaye, interim CEO of Sarepta Therapeutics with an award for their commitment to the Duchenne community.
“We are delighted to honor Dr. Ed Kaye and the entire Sarepta Therapeutics organization for their tireless efforts in what potentially could be the first Duchenne drug approved in the U.S.,” said Paul Miller, co-founder of CureDuchenne. “Their Duchenne drug has been beneficial for those who have been on the clinical trials. In addition to their expertise in rare disease Ed brings commitment and compassion for the patients.”
“Monday, April 25th was a historic day for Duchenne Muscular Dystrophy, there were hundreds of families who came to Washington D.C. to support the boys who were receiving eteplirsen,” stated Ed Kaye, interim CEO of Sarepta Therapeutics. “We were so proud of the boys that came together, and what you need to remember is that everyone is behind this community. This is not a single step, this is a long journey.”
BLINGO is spearheaded by the Gambhir family, who reside in Center City Philadelphia and know first-hand the impact that Duchenne can have on a young boy and his family. They are devoted to finding a treatment and a cure for their son Yuva, a spirited 14-year-old who gave a heartfelt and inspiring speech explaining his lack of ability to stand, walk and keep up with his friends.
“Since last year, Duchenne has made things even more difficult for me and my muscles are even weaker,” stated Yuva Gambhir. “I don’t want to focus on these negative things because in the middle of all of this craziness I realized something, I see how the doctors and everyone else really go out of their way to help me and other boys with Duchenne. I think we can all be the change we wish to see, we just have to look past the challenges.”
CureDuchenne uses a multi-dimensional approach to identifying and funding potential treatments for Duchenne. CureDuchenne has been working to “treat the whole disease,” with multiple therapeutic strategies working together to address the many effects that Duchenne has on the body. CureDuchenne has funded research for exon skipping, anti-inflammatory and anti-fibrotic treatments, gene therapy, as well as cardiac drugs to treat heart failure, the leading cause of death for those with Duchenne.
BLINGO to CureDuchenne sponsors included: Allan Domb Real Estate, Baker Hostetler, CA Spalding & Co, the Condo Shop, Domus Inc., Drexel University, Sandra Duggan and Javier Kuehnle, EKL Machine, The Tax Accounting Group of Duane Morris LLP, Gambhir Family, Governor’s Woods Foundation, Fatema and Iqbal Haider, KGM, Krevitz Metals, Oller Family, OTG Management, Parx Casino, Philadelphia Eagles, Photobot, Sarepta Therapeutics, Small Luxury Hotels of the World, Sugar House Casino, Thomas Jefferson University and Jefferson Health, and Wescott Financial Advisory Group LLC.
CureDuchenne is a national nonprofit organization located in Newport Beach, Calif., dedicated to finding a cure for Duchenne, the most common and most lethal form of muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 boys worldwide. CureDuchenne has garnered international attention for its efforts to raise funds and awareness for Duchenne through venture philanthropy. With the help of CureDuchenne’s distinguished international panel of Scientific Advisors, funds raised by CureDuchenne support the most promising research aimed at treating and curing Duchenne. To date, nine CureDuchenne research projects have advanced into human clinical trials – a unique accomplishment, as few health-related nonprofits have been as successful in being a catalyst for human clinical trials. www.cureduchenne.org
Duchenne is a devastating muscle disease in children. Historically, most boys who have it do not survive beyond their mid-twenties, and those that do will be wheelchair bound by age 12 and experience social isolation. The simplest of tasks become difficult, and in the later stages, heart and breathing muscles begin to fail. Nearly 20,000 boys are living with the disease in the United States alone and more than 300,000 worldwide. The disorder knows no cultural, economic or social boundaries.