“This recognition from the White House shows that Emily’s Entourage is leading the way—pushing the boundaries of science and helping to write the story of precision medicine,” said Emily. “Precision medicine is not just a bold political initiative or exciting new era of biomedical research and personalized therapeutics—it’s my hope for a future.”
“Our goal is to find therapies that are designed for even those of us with rare mutations, so that no one has a mutation that’s too uncommon to get on the research radar. This is the fight to save my life and the lives of other people with rare mutations like mine,” said Emily, describing why Emily’s Entourage (EE) has raised $1.5 million since 2011 to fund research on nonsense CF mutations.
The 2012 approval of the breakthrough drug Kalydeco was a wakeup call for Emily and her Entourage. The first drug to treat the root cause of CF, Kalydeco has been a game-changer for 4 percent of the CF population that shares one common mutation. Emily has watched friends move off lung transplant wait lists, begin families, run marathons, and start planning a future they never thought they would have—all thanks to Kalydeco. Unfortunately, it does not help Emily’s rare CF mutation.
Another CF breakthrough occurred July 2nd when the FDA approved a combination therapy that will help approximately 50 percent of the CF population—but again, not Emily. “It’s awesome to see my friends get these breakthroughs. But we want a breakthrough like that for me and everyone else with CF too,” she said.
Emily was among the nine White House “Champions of Change” introduced by NIH Director Francis Collins, MD, PhD, who said, “Our cause is to change the way medicine is practiced by taking into account individual differences.”
The President’s Precision Medicine Initiative aims to build a cohort of one million individuals by year-end to expedite the pace of discovery for medical research and therapeutics targeted precisely to individuals—including people like Emily with rare, advanced, and life-threatening diseases who are in a race against time.
This honor and the opportunity to speak at the White House are moments that Emily will cherish forever. Her story resonates at a policy and personal level as reflected in the widespread media coverage of her honor on CBS3, NBC10, CBS3 Local, Fox29, and in the Philadelphia Inquirer, the Jewish Exponent, the Philly Voice, and more.
Read a blog post Emily wrote for the White House website
Watch a video of the Champions of Change event (Emily’s panel begins at 0:26)