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Washington, D.C., September 30, 2016 – Today, President Obama signed the Advancing Hope Act (S. 1878), extending the Rare Pediatric Disease Priority Review Voucher (PRV) program until December 31.
“On behalf of the 15 million children with rare diseases, we thank the President for signing the Advancing Hope Act so… Read More
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A recent article in the Financial Times (June 24, 2016) looked at the growing impact families have on medical research. According to the Center of Strategic Philanthropy at the Milken Institute, family foundations provide approximately 3 percent of all medical research funding in the U.S. Families also help bridge… Read More
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When he was six years old, Arthur Caplan came down with polio and was temporarily paralyzed. That episode – which included time at Boston’s Mass General Hospital followed by rehab – “always left me with a patient perspective,” Dr. Caplan says.
That perspective and his tendency to be drawn to complex issues have led him… Read More
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A leading expert in her field, Dawn Laney, M.S., C.G.C., C.C.R.C. is a genetic counselor, instructor, and program leader at Emory University in the Department of Human Genetics. She manages Emory’s infusion center and lysosome storage disease clinical research, and is a sought-after specialist in Fabry Disease, a rare genetic disorder that can lead to kidney failure,… Read More
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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… Read More
