PHILADELPHIA--(BUSINESS WIRE)--CureDuchenne, a national nonprofit that raises awareness and funds research to find a cure for Duchenne Muscular Dystrophy, will put the bling in bingo for their third annual fundraiser on Saturday, April 30th, 2016 at The Logan Hotel. BLINGO to CureDuchenne is a vibrant evening, featuring FOX29 News General Assignment Reporter, Jennaphr Frederick as the MC. Alongside, former Governor Edward G. Rendell, WMMR Preston and Steve show producer, Marisa Magnatta, local sensation John Bolaris, DJ Nigel Richards and comedian Joe Conklin in celebration for a cause to help save the lives of those with Duchenne.
Duchenne is the most common and lethal form of muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 boys worldwide with nearly 20,000 boys living with the disease in the United States alone. Boys are usually diagnosed by age five, lose their ability to walk by 12 and most don’t survive their mid-20s. Currently, there is no treatment or cure for Duchenne.
BLINGO is spearheaded by the Gambhir family, who reside in Center City 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 is battling the illness that has made it difficult for him to stand, walk and even reach.
“Our goal is to increase awareness of this disease and raise funds to find a cure,” stated Yuva’s father, Manu. “My family and I have been through many trials and we continue to stay positive in hopes that one day there will be approval of the experimental drug, eteplirsen, helping slow the progression of the disease.”
This year, BLINGO to CureDuchenne will honor Sarepta Therapeutics, a biopharmaceutical company focused on developing innovative RNA-targeted therapeutics, as a supporter in fighting Duchenne muscular dystrophy. Sarepta Therapeutics recently announced that the Peripheral and Central Nervous System (PCNS) Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review Sarepta’s New Drug Application (NDA) for eteplirsen, an experimental drug currently in clinical trials, on April 25, 2016. The FDA PDUFA date scheduled for completion of the review is May 26, 2016. Yuva is currently on a Sarepta trial and his father, Manu, will be attending the review.
CureDuchenne pushes toward innovation and advancement of Duchenne research. CureDuchenne provided early funding to Sarepta which helped the company move into human clinical trials. 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.
Tickets for BLINGO to CureDuchene and sponsorship information can be found at www.blingophilly.org.
BLINGO to CureDuchenne sponsors include: 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.